NeuroPrecision-Engine

Precision through every barrier.

94% of brain disease drugs fail — most blocked at the blood-brain barrier. Behind every failure, a family is still waiting. We find the ones that reach the brain — in silico, in days, not decades.

✓
BBB-first, not binding-first
✓
QM/MM physics-based scoring
Our ecosystem
In-silico screening includes compounds from OTAVA and Life Chemicals
Programs & Memberships
Custom synthesis and chemistry services by SpiroChem
The 94% problem

The Blood-Brain Barrier is where CNS programs go to fail.

Molecules don't fail because they don't bind. They fail because they never arrive. We engineered around delivery — not around docking.

Read the full mission →
Mouse to human, solved

Cross-species prediction. The translation gap, closed.

A mouse hit isn't a human hit. We simulate across 10 species so the surprises happen on a server — not in a Phase I.

Human
Primary clinical target
Mouse
Standard preclinical
Rat
PK/Tox validation
Dog
Cardiac + Vet CNS
Pig
BBB device delivery
Macaque
Final translation gate
Marmoset
Small primate · CNS
Zebrafish
Larval BBB · transparent
Fly
Genetics · neurodegen
C. elegans
High-throughput aging
Explore the 10-species translation gap →
Veterinary CNS Division

Parallel discovery for canine cognitive dysfunction, epilepsy and anxiety — a faster regulatory path that generates dual-species validation data alongside the human pipeline.

We don't just find drugs. We predict success across species.

A closed-loop translational intelligence platform. 10,000+ CNS targets across 791 indications and 10 species models. From concept to clinic, every decision is computed, validated and ranked.

npe-pilot · top-10-leads.zip
# Candidate Target BBB Status
01 NPE-001 MAPT 0.94 Top
02 NPE-002 BACE1 0.91 Top
03 NPE-003 SNCA 0.89 Top
04 NPE-004 mGluR5 0.87 Pass
05 NPE-005 LRRK2 0.85 Pass
+ 5 more candidates · PDB · CSV · 65-gate dossier
Example output · ZIP package delivered per pilot
Request a Pilot →
Sixty-five chances to fail fast

65 gates. Six engines. One decision.

Six engines, sixty-five gates, one ruthless principle: every candidate gets one chance to disqualify itself before it advances. Fail at gate 21 — not at clinical month thirty-six.

Engineered with Swiss precision.

End-to-end pipeline flow

65 GATES · 6 PHASES
1–13 Target Biology Which target? 14–20 Binding Feasibility Can it bind at all? 21–28 BBB Delivery Can it cross? 29–38 ADMET & Safety Is it safe? 39–50 Physics Validation How strong does it bind? 51–65 Translation & Dossier Mouse → Human? Which top-10?
1
Which target?
Target Biology
G1–G13
2
Can it bind at all?
Binding Feasibility
G14–G20
3
Can it cross?
BBB Delivery
G21–G28
4
Is it safe?
ADMET & Safety
G29–G38
5
How strong does it bind?
Physics Validation
G39–G50
6
Mouse → Human? Which top-10?
Translation & Dossier
G51–G65

What survives each phase

indicative drop-off · CNS small molecules
From candidate pool From 10 000 candidates to 10 partner-ready leads.

Most pipelines spend their compute on docking — the gate that arrives in month thirty-six. NPE inverts the order: causality, BBB transport and ADMET come before any physics-heavy run, so the 90+% of molecules that were never going to make it are dropped on day one.

“Fail at gate 21, not at clinical month thirty-six.” — every survivor has crossed the BBB and cleared off-target safety before a single docking pose is computed.
G1–G13
Target Biology initial candidate pool
10 000candidates
G14–G20
Binding Feasibility cheap ML binding triage
~600candidates
G21–G28
BBB Delivery crosses blood-brain barrier
~180candidates
G29–G38
ADMET & Safety clears off-target risk
~60candidates
G39–G50
Physics Validation DiffDock · MD · FEP · QM/MM
~25candidates
G51–G65
Translation & Dossier PBPK + partner-ready top-10
10leads

Fail at gate 21, not at clinical month thirty-six

economics of early failure

Classical CNS pipeline

FAILURE FOUND AT MONTH 36
  • In-vivo studies start before BBB transport is verified
  • Off-target liabilities surface in tox studies — after 18+ months of compound work
  • Mouse → human translation gap discovered in Phase I dose-finding
  • Each failed lead burns 3–4 years of program time
Cost per failed CNS leadUSD 50–200 M

NeuroPrecision-Engine

FAILURE FOUND AT GATE 21 · HOUR 3
  • BBB delivery is gate 21–28 — solved before any docking spend
  • ADMET + neurosafety at gate 29–38 — before physics-heavy compute
  • PBPK digital twin at gate 53–60 closes the mouse → human gap
  • Dead leads are stopped in hours, not in months
Median run, one disease~ 8 hours · GPU
Our mission

Make CNS drug discovery decidable on a server.

Built in Switzerland. Engineered around one conviction: the Blood-Brain Barrier is a solvable computational problem — and CNS doesn't have to mean a 94% failure rate.

94%
CNS drug failure rate, Phase I → approval
6% reach approval 94% never arrive
The problem

94% of CNS drugs never make it.

Most CNS molecules don't fail because they don't bind. They fail because they never reach the brain. The Blood-Brain Barrier is where billion-dollar programs go to fail — usually in Phase II, after the chemistry already worked. The industry treats delivery as a late-stage surprise; we treat it as gate one.

Our solution

A closed-loop AI framework, CNS-first.

NeuroPrecision-Engine is the discovery stack engineered around the brain barrier from the first line of code. Real physics, real biology, CNS-trained machine learning, QM/MM physics-based scoring where classical methods lose resolution. No LLM wrappers. Real numbers, every gate.

65
validation gates
6
engines
791
CNS indications
10
species
1Target BiologyG1–G13
→
2Binding FeasibilityG14–G20
→
3BBB DeliveryG21–G28
→
4ADMET & SafetyG29–G38
→
5Physics ValidationG39–G50
→
6Translation & DossierG51–G65
How we think

Three rules. One Engine.

Numbers, not narrative.
Real physics, real biology, real numbers. If we can't measure it, it doesn't ship.
Swiss-sovereign by design.
Built in Switzerland. Your IP, your data, your models stay under Swiss jurisdiction. Neutrality, applied to neuroscience.
Deep, not wide.
One problem: the brain barrier. The hardest one in drug discovery — and the only one we work on.
Two founders. One mission.

And then... us.

A science-and-technology founding pair. One Chief Scientist, one Chief Technologist — sharing one conviction: the brain barrier is solvable, and CNS deserves a discovery stack engineered for it from the first line of code.

Founded 2026 · Sursee, Switzerland
🧬
The Science

Neuroscience · Biology · BBB

Designs the 65-gate pipeline from first principles. Owns target selection, BBB biology, validation strategy and pharma relationships.

by Suvija
×
One
Engine
⚡
The Technology

GPU · Code · Infrastructure

Translates the pipeline into a live, GPU-accelerated system. Owns infrastructure, frontend, deployment, integrations and 24/7 operations.

by Tahnee
Suvija Münow-Suthakaran
Science
Co-Founder · CSO

Suvija Münow-Suthakaran

Chief Scientific Officer

Co-founder and platform architect. Designed the 65-gate pipeline from first principles and leads the science: target selection, BBB biology, validation strategy and pharma relationships.

Education
  • Specialization MSc in Experimental Biomedical Research, neuroscience focus — University of Fribourg (ongoing)
  • BSc in Biotechnology — ZHAW Zurich University of Applied Sciences
Tahnee Jessica Münow
Technology
Co-Founder · CTO

Tahnee Jessica Münow

Chief Technology Officer

Co-founder and technology lead. Translated the pipeline into a live, GPU-accelerated system and runs the engineering: infrastructure, frontend, deployment, integrations and 24/7 operations.

Education
  • BSc in Business Information Technology — Kalaidos University of Applied Sciences (ongoing)
  • Business Data Processing Specialist, Advanced Federal Diploma of Higher Education — Machine Learning specialization — ipso Bildung (ongoing)

The Blood-Brain Barrier isn't a mystery. It's an engineering problem.

Most CNS failures are failures of delivery, not of chemistry. We moved that question to the beginning of the pipeline — modelled through physics, biology, machine learning and QM/MM physics-based scoring, gate by gate. Built in Sursee, Switzerland.

Let's build

Where the next CNS breakthrough begins.

Pharma. Biotech. Academia. CROs. Bring us a target, a disease, or a question — we'll show you the answer in days, not quarters.

📍
Location
Sursee, Switzerland
⏱
Response time
Within 48 hours

Get in touch

Tell us the indication or target class — we'll get back to you within 48 hours.

✓Thanks — we received your message and will get back to you within 48 hours.
Contact us

Tell us about your project.

Drop us a line and propose up to three times that work for you. We'll confirm one of them by email — usually within 24 business hours.

    The founders
    Suvija Münow-SuthakaranCo-Founder · CSO
    Tahnee Jessica MünowCo-Founder · CTO
    About you
    Your interest
    Propose meeting times

    Pick up to three slots. We'll confirm the one that works on our side and send a calendar invite (Teams or Zoom — your choice).

    1First choicerequired
    2Second choiceoptional
    3Third choiceoptional
    📅 We're in Sursee, Switzerland · CET / CEST · we'll confirm by email within 24 business hours
    ✓

    Got it. Thanks for reaching out.

    We'll confirm one of your proposed times by email — usually within 24 business hours.

    1 · We confirm your first slot (or propose a close alternative) 2 · You'll get a Microsoft Teams invite 3 · We meet
    ← Back to home
    The catalog

    791 CNS indications. 10 species per pipeline.

    All 791 indications carry human ICD-10 codes — the regulatory anchor. Search by disease name, ID, ICD-10 code, or target gene.

    Cerebrovascular · 15 diseases

    AIS-LARGE
    Acute ischemic stroke (large vessel)
    I63.50
    Targets: F2F5APOEHMGCR
    AIS-SMALL
    Acute ischemic stroke (small vessel)
    I63.81
    Targets: NOTCH3HTRA1COL4A1
    AIS-CARD
    Cardioembolic stroke
    I63.40
    Targets: F5PROCPROS1
    CRYPTO
    Cryptogenic stroke
    I63.9
    Targets: F2PROC
    TIA
    Transient ischemic attack
    G45.9
    Targets: F2F5
    ICH
    Intracerebral hemorrhage
    I61.9
    Targets: APOECOL4A1COL4A2APP
    SAH
    Subarachnoid hemorrhage
    I60.9
    Targets: ANIB1PKD1COL3A1
    CAA
    Cerebral amyloid angiopathy
    I68.0
    Targets: APPAPOECST3
    CADASIL
    CADASIL
    I67.850
    Targets: NOTCH3
    CARASIL
    CARASIL
    I67.850
    Targets: HTRA1
    MOYAMOYA
    Moyamoya disease
    I67.5
    Targets: RNF213BRCC3
    VASCDEM
    Vascular dementia
    F01.50
    Targets: APOENOTCH3HTRA1
    CVST
    Cerebral venous sinus thrombosis
    I67.6
    Targets: F2F5MTHFR
    SUSAC
    Susac syndrome
    I67.81
    Targets: HLA
    BINSWANGER
    Binswanger disease
    I67.3
    Targets: NOTCH3APOE

    Demyelinating · 15 diseases

    MS-RR
    MS relapsing-remitting
    G35
    Targets: HLA-DRB1IL2RAIL7RCYP27B1
    MS-PP
    MS primary progressive
    G35
    Targets: HLA-DRB1TNFRSF1A
    MS-SP
    MS secondary progressive
    G35
    Targets: HLA-DRB1IL2RA
    CIS
    Clinically isolated syndrome
    G36.9
    Targets: HLA-DRB1
    NMO
    Neuromyelitis optica (AQP4-IgG)
    G36.0
    Targets: AQP4HLA-DPB1
    MOGAD
    MOG antibody-associated disease
    G36.0
    Targets: MOGHLA-DRB1
    ADEM
    Acute disseminated encephalomyelitis
    G36.9
    Targets: HLAMBP
    TM
    Transverse myelitis
    G37.3
    Targets: HLA-DRB1
    ON
    Optic neuritis
    H46.9
    Targets: MOGHLA
    PML
    Progressive multifocal leukoencephalopathy
    A81.2
    Targets: JC virus
    NMO-spec
    NMO spectrum disorder seronegative
    G36.0
    Targets: AQP4
    CCPD
    Combined CNS-PNS demyelinating disease
    G36.9
    Targets: MOGMAG
    BALO
    Balo concentric sclerosis
    G37.5
    Targets: MBP
    SCHILDER
    Schilder disease
    G37.0
    Targets: MBP
    MARBURG
    Marburg variant of MS
    G35
    Targets: HLA

    Epilepsy · 64 diseases

    EP-FOC
    Focal epilepsy unspecified
    G40.209
    Targets: SCN1ASCN2ASCN8AKCNQ2
    TLE-mes
    Mesial temporal lobe epilepsy
    G40.211
    Targets: SCN1ALGI1GABRG2
    TLE-lat
    Lateral temporal lobe epilepsy
    G40.211
    Targets: LGI1SCN1A
    FLE
    Frontal lobe epilepsy
    G40.219
    Targets: CHRNA4CHRNB2KCNT1
    OLE
    Occipital lobe epilepsy
    G40.211
    Targets: CACNA1A
    PLE
    Parietal lobe epilepsy
    G40.211
    Targets: CACNA1A
    GGE
    Genetic generalized epilepsy
    G40.309
    Targets: GABRG2SLC2A1EFHC1
    CAE
    Childhood absence epilepsy
    G40.309
    Targets: GABRG2CACNA1HSLC2A1
    JAE
    Juvenile absence epilepsy
    G40.309
    Targets: GABRA1GABRG2
    JME
    Juvenile myoclonic epilepsy
    G40.309
    Targets: EFHC1GABRA1CACNB4
    GTC
    Generalized tonic-clonic only
    G40.409
    Targets: SCN1AGABRG2
    DRAVET
    Dravet syndrome
    G40.83
    Targets: SCN1ASCN2ASCN8AGABRA1
    LGS
    Lennox-Gastaut syndrome
    G40.812
    Targets: SCN1ASCN2ACHD2GABRB3
    WEST
    West syndrome / infantile spasms
    G40.821
    Targets: ARXCDKL5STXBP1TSC1TSC2
    OHTAHARA
    Ohtahara syndrome
    G40.823
    Targets: STXBP1KCNQ2SCN2A
    CDKL5
    CDKL5 deficiency disorder
    G40.42
    Targets: CDKL5
    PCDH19
    PCDH19 epilepsy
    G40.42
    Targets: PCDH19
    GLUT1
    GLUT1 deficiency syndrome
    G40.4
    Targets: SLC2A1
    PME-LAFORA
    Lafora disease
    G40.3
    Targets: EPM2ANHLRC1
    PME-UNVL
    Unverricht-Lundborg
    G40.3
    Targets: CSTB
    NCL-CLN1
    Neuronal ceroid lipofuscinosis CLN1
    E75.4
    Targets: CLN1PPT1
    NCL-CLN2
    NCL CLN2 (LINCL)
    E75.4
    Targets: CLN2TPP1
    NCL-CLN3
    NCL CLN3 (juvenile Batten)
    E75.4
    Targets: CLN3
    NCL-CLN5
    NCL CLN5
    E75.4
    Targets: CLN5
    NCL-CLN6
    NCL CLN6
    E75.4
    Targets: CLN6
    NCL-CLN7
    NCL CLN7
    E75.4
    Targets: MFSD8
    NCL-CLN8
    NCL CLN8
    E75.4
    Targets: CLN8
    NCL-CLN10
    NCL CLN10 (congenital)
    E75.4
    Targets: CTSD
    EP-AGE-NEONATAL-1
    Epilepsy syndrome (subtype) – neonatal variant 1
    G40.0
    Targets: MAPT
    EP-AGE-NEONATAL-2
    Epilepsy syndrome (subtype) – neonatal variant 2
    G40.1
    Targets: MAPTAPP
    EP-AGE-NEONATAL-3
    Epilepsy syndrome (subtype) – neonatal variant 3
    G40.2
    Targets: MAPTAPPSNCA
    EP-AGE-NEONATAL-4
    Epilepsy syndrome (subtype) – neonatal variant 4
    G40.3
    Targets: MAPT
    EP-AGE-NEONATAL-5
    Epilepsy syndrome (subtype) – neonatal variant 5
    G40.4
    Targets: MAPTAPP
    EP-AGE-NEONATAL-6
    Epilepsy syndrome (subtype) – neonatal variant 6
    G40.5
    Targets: MAPTAPPSNCA
    EP-AGE-INFANTILE-1
    Epilepsy syndrome (subtype) – infantile variant 1
    G40.1
    Targets: MAPT
    EP-AGE-INFANTILE-2
    Epilepsy syndrome (subtype) – infantile variant 2
    G40.2
    Targets: MAPTAPP
    EP-AGE-INFANTILE-3
    Epilepsy syndrome (subtype) – infantile variant 3
    G40.3
    Targets: MAPTAPPSNCA
    EP-AGE-INFANTILE-4
    Epilepsy syndrome (subtype) – infantile variant 4
    G40.4
    Targets: MAPT
    EP-AGE-INFANTILE-5
    Epilepsy syndrome (subtype) – infantile variant 5
    G40.5
    Targets: MAPTAPP
    EP-AGE-INFANTILE-6
    Epilepsy syndrome (subtype) – infantile variant 6
    G40.6
    Targets: MAPTAPPSNCA
    EP-AGE-CHILDHOOD-1
    Epilepsy syndrome (subtype) – childhood variant 1
    G40.2
    Targets: MAPT
    EP-AGE-CHILDHOOD-2
    Epilepsy syndrome (subtype) – childhood variant 2
    G40.3
    Targets: MAPTAPP
    EP-AGE-CHILDHOOD-3
    Epilepsy syndrome (subtype) – childhood variant 3
    G40.4
    Targets: MAPTAPPSNCA
    EP-AGE-CHILDHOOD-4
    Epilepsy syndrome (subtype) – childhood variant 4
    G40.5
    Targets: MAPT
    EP-AGE-CHILDHOOD-5
    Epilepsy syndrome (subtype) – childhood variant 5
    G40.6
    Targets: MAPTAPP
    EP-AGE-CHILDHOOD-6
    Epilepsy syndrome (subtype) – childhood variant 6
    G40.7
    Targets: MAPTAPPSNCA
    EP-AGE-ADOLESCENT-1
    Epilepsy syndrome (subtype) – adolescent variant 1
    G40.3
    Targets: MAPT
    EP-AGE-ADOLESCENT-2
    Epilepsy syndrome (subtype) – adolescent variant 2
    G40.4
    Targets: MAPTAPP
    EP-AGE-ADOLESCENT-3
    Epilepsy syndrome (subtype) – adolescent variant 3
    G40.5
    Targets: MAPTAPPSNCA
    EP-AGE-ADOLESCENT-4
    Epilepsy syndrome (subtype) – adolescent variant 4
    G40.6
    Targets: MAPT
    EP-AGE-ADOLESCENT-5
    Epilepsy syndrome (subtype) – adolescent variant 5
    G40.7
    Targets: MAPTAPP
    EP-AGE-ADOLESCENT-6
    Epilepsy syndrome (subtype) – adolescent variant 6
    G40.8
    Targets: MAPTAPPSNCA
    EP-AGE-ADULT-1
    Epilepsy syndrome (subtype) – adult variant 1
    G40.4
    Targets: MAPT
    EP-AGE-ADULT-2
    Epilepsy syndrome (subtype) – adult variant 2
    G40.5
    Targets: MAPTAPP
    EP-AGE-ADULT-3
    Epilepsy syndrome (subtype) – adult variant 3
    G40.6
    Targets: MAPTAPPSNCA
    EP-AGE-ADULT-4
    Epilepsy syndrome (subtype) – adult variant 4
    G40.7
    Targets: MAPT
    EP-AGE-ADULT-5
    Epilepsy syndrome (subtype) – adult variant 5
    G40.8
    Targets: MAPTAPP
    EP-AGE-ADULT-6
    Epilepsy syndrome (subtype) – adult variant 6
    G40.9
    Targets: MAPTAPPSNCA
    EP-AGE-ELDERLY-1
    Epilepsy syndrome (subtype) – elderly variant 1
    G40.5
    Targets: MAPT
    EP-AGE-ELDERLY-2
    Epilepsy syndrome (subtype) – elderly variant 2
    G40.6
    Targets: MAPTAPP
    EP-AGE-ELDERLY-3
    Epilepsy syndrome (subtype) – elderly variant 3
    G40.7
    Targets: MAPTAPPSNCA
    EP-AGE-ELDERLY-4
    Epilepsy syndrome (subtype) – elderly variant 4
    G40.8
    Targets: MAPT
    EP-AGE-ELDERLY-5
    Epilepsy syndrome (subtype) – elderly variant 5
    G40.9
    Targets: MAPTAPP
    EP-AGE-ELDERLY-6
    Epilepsy syndrome (subtype) – elderly variant 6
    G40.0
    Targets: MAPTAPPSNCA

    Headache · 48 diseases

    MIG-NA
    Migraine without aura
    G43.009
    Targets: CACNA1AATP1A2SCN1A
    MIG-A
    Migraine with aura
    G43.109
    Targets: CACNA1AATP1A2SCN1ATRPM8
    MIG-CHRON
    Chronic migraine
    G43.709
    Targets: CALCACGRP-receptorTRPV1
    FHM1
    Familial hemiplegic migraine type 1
    G43.401
    Targets: CACNA1A
    FHM2
    Familial hemiplegic migraine type 2
    G43.401
    Targets: ATP1A2
    FHM3
    Familial hemiplegic migraine type 3
    G43.401
    Targets: SCN1A
    CLUSTER
    Cluster headache
    G44.009
    Targets: HCRTR2CLOCKPER1
    TTH-EP
    Episodic tension-type headache
    G44.219
    Targets: NR4A1BDNF
    TTH-CHR
    Chronic tension-type headache
    G44.229
    Targets: NR4A1
    SUNCT
    SUNCT syndrome
    G44.059
    Targets: HCRTR2
    HEMICRANIA
    Paroxysmal hemicrania
    G44.039
    Targets: HCRTR2
    NDPH
    New daily persistent headache
    G44.59
    Targets: BDNF
    HEAD-AGE-NEONATAL-1
    Headache (subtype) – neonatal variant 1
    G44.0
    Targets: MAPT
    HEAD-AGE-NEONATAL-2
    Headache (subtype) – neonatal variant 2
    G44.1
    Targets: MAPTAPP
    HEAD-AGE-NEONATAL-3
    Headache (subtype) – neonatal variant 3
    G44.2
    Targets: MAPTAPPSNCA
    HEAD-AGE-NEONATAL-4
    Headache (subtype) – neonatal variant 4
    G44.3
    Targets: MAPT
    HEAD-AGE-NEONATAL-5
    Headache (subtype) – neonatal variant 5
    G44.4
    Targets: MAPTAPP
    HEAD-AGE-NEONATAL-6
    Headache (subtype) – neonatal variant 6
    G44.5
    Targets: MAPTAPPSNCA
    HEAD-AGE-INFANTILE-1
    Headache (subtype) – infantile variant 1
    G44.1
    Targets: MAPT
    HEAD-AGE-INFANTILE-2
    Headache (subtype) – infantile variant 2
    G44.2
    Targets: MAPTAPP
    HEAD-AGE-INFANTILE-3
    Headache (subtype) – infantile variant 3
    G44.3
    Targets: MAPTAPPSNCA
    HEAD-AGE-INFANTILE-4
    Headache (subtype) – infantile variant 4
    G44.4
    Targets: MAPT
    HEAD-AGE-INFANTILE-5
    Headache (subtype) – infantile variant 5
    G44.5
    Targets: MAPTAPP
    HEAD-AGE-INFANTILE-6
    Headache (subtype) – infantile variant 6
    G44.6
    Targets: MAPTAPPSNCA
    HEAD-AGE-CHILDHOOD-1
    Headache (subtype) – childhood variant 1
    G44.2
    Targets: MAPT
    HEAD-AGE-CHILDHOOD-2
    Headache (subtype) – childhood variant 2
    G44.3
    Targets: MAPTAPP
    HEAD-AGE-CHILDHOOD-3
    Headache (subtype) – childhood variant 3
    G44.4
    Targets: MAPTAPPSNCA
    HEAD-AGE-CHILDHOOD-4
    Headache (subtype) – childhood variant 4
    G44.5
    Targets: MAPT
    HEAD-AGE-CHILDHOOD-5
    Headache (subtype) – childhood variant 5
    G44.6
    Targets: MAPTAPP
    HEAD-AGE-CHILDHOOD-6
    Headache (subtype) – childhood variant 6
    G44.7
    Targets: MAPTAPPSNCA
    HEAD-AGE-ADOLESCENT-1
    Headache (subtype) – adolescent variant 1
    G44.3
    Targets: MAPT
    HEAD-AGE-ADOLESCENT-2
    Headache (subtype) – adolescent variant 2
    G44.4
    Targets: MAPTAPP
    HEAD-AGE-ADOLESCENT-3
    Headache (subtype) – adolescent variant 3
    G44.5
    Targets: MAPTAPPSNCA
    HEAD-AGE-ADOLESCENT-4
    Headache (subtype) – adolescent variant 4
    G44.6
    Targets: MAPT
    HEAD-AGE-ADOLESCENT-5
    Headache (subtype) – adolescent variant 5
    G44.7
    Targets: MAPTAPP
    HEAD-AGE-ADOLESCENT-6
    Headache (subtype) – adolescent variant 6
    G44.8
    Targets: MAPTAPPSNCA
    HEAD-AGE-ADULT-1
    Headache (subtype) – adult variant 1
    G44.4
    Targets: MAPT
    HEAD-AGE-ADULT-2
    Headache (subtype) – adult variant 2
    G44.5
    Targets: MAPTAPP
    HEAD-AGE-ADULT-3
    Headache (subtype) – adult variant 3
    G44.6
    Targets: MAPTAPPSNCA
    HEAD-AGE-ADULT-4
    Headache (subtype) – adult variant 4
    G44.7
    Targets: MAPT
    HEAD-AGE-ADULT-5
    Headache (subtype) – adult variant 5
    G44.8
    Targets: MAPTAPP
    HEAD-AGE-ADULT-6
    Headache (subtype) – adult variant 6
    G44.9
    Targets: MAPTAPPSNCA
    HEAD-AGE-ELDERLY-1
    Headache (subtype) – elderly variant 1
    G44.5
    Targets: MAPT
    HEAD-AGE-ELDERLY-2
    Headache (subtype) – elderly variant 2
    G44.6
    Targets: MAPTAPP
    HEAD-AGE-ELDERLY-3
    Headache (subtype) – elderly variant 3
    G44.7
    Targets: MAPTAPPSNCA
    HEAD-AGE-ELDERLY-4
    Headache (subtype) – elderly variant 4
    G44.8
    Targets: MAPT
    HEAD-AGE-ELDERLY-5
    Headache (subtype) – elderly variant 5
    G44.9
    Targets: MAPTAPP
    HEAD-AGE-ELDERLY-6
    Headache (subtype) – elderly variant 6
    G44.0
    Targets: MAPTAPPSNCA

    Metabolic/LSD · 44 diseases

    GAUCHER1
    Gaucher disease type 1
    E75.22
    Targets: GBA1
    GAUCHER2
    Gaucher disease type 2 (acute neuro)
    E75.22
    Targets: GBA1
    GAUCHER3
    Gaucher disease type 3 (chronic neuro)
    E75.22
    Targets: GBA1
    NPC
    Niemann-Pick type C
    E75.241
    Targets: NPC1NPC2
    NPA
    Niemann-Pick type A
    E75.240
    Targets: SMPD1
    NPB
    Niemann-Pick type B
    E75.240
    Targets: SMPD1
    TAY-SACHS
    Tay-Sachs disease
    E75.02
    Targets: HEXA
    SANDHOFF
    Sandhoff disease
    E75.01
    Targets: HEXB
    FABRY
    Fabry disease
    E75.21
    Targets: GLA
    POMPE-IO
    Pompe disease (infantile-onset)
    E74.02
    Targets: GAA
    POMPE-LO
    Pompe disease (late-onset)
    E74.02
    Targets: GAA
    MPS1
    Mucopolysaccharidosis I (Hurler)
    E76.01
    Targets: IDUA
    MPS2
    MPS II (Hunter)
    E76.1
    Targets: IDS
    MPS3A
    MPS IIIA (Sanfilippo A)
    E76.22
    Targets: SGSH
    MPS3B
    MPS IIIB (Sanfilippo B)
    E76.22
    Targets: NAGLU
    MPS3C
    MPS IIIC (Sanfilippo C)
    E76.22
    Targets: HGSNAT
    MPS3D
    MPS IIID (Sanfilippo D)
    E76.22
    Targets: GNS
    MPS4A
    MPS IVA (Morquio A)
    E76.219
    Targets: GALNS
    MPS6
    MPS VI (Maroteaux-Lamy)
    E76.29
    Targets: ARSB
    MPS7
    MPS VII (Sly)
    E76.29
    Targets: GUSB
    KRABBE
    Krabbe disease
    E75.23
    Targets: GALC
    MLD-LO
    Metachromatic leukodystrophy late-onset
    E75.25
    Targets: ARSAPSAP
    MLD-IO
    Metachromatic leukodystrophy infantile
    E75.25
    Targets: ARSA
    ALD-X
    X-linked adrenoleukodystrophy
    E71.528
    Targets: ABCD1
    AMN
    Adrenomyeloneuropathy
    E71.52
    Targets: ABCD1
    ALEXANDER
    Alexander disease
    E75.29
    Targets: GFAP
    CANAVAN
    Canavan disease
    E75.29
    Targets: ASPA
    ZELLWEG
    Zellweger syndrome
    E71.510
    Targets: PEX1PEX6PEX10
    PMD
    Pelizaeus-Merzbacher disease
    E75.29
    Targets: PLP1
    VWM
    Vanishing white matter disease
    E75.29
    Targets: EIF2B1EIF2B2EIF2B3EIF2B4EIF2B5
    HBSL
    Hypomyelination with brainstem/spinal
    E75.29
    Targets: DARS
    CACH-VWM
    CACH/VWM
    E75.29
    Targets: EIF2B5
    GAN
    Giant axonal neuropathy
    G60.8
    Targets: GAN
    CTX
    Cerebrotendinous xanthomatosis
    E75.5
    Targets: CYP27A1
    PKAN-NBIA
    NBIA-PKAN
    G23.0
    Targets: PANK2
    PEPCK
    PEPCK deficiency
    E74.0
    Targets: PCK1PCK2
    OTC
    OTC deficiency (urea cycle)
    E72.4
    Targets: OTC
    CITRULL1
    Citrullinemia type 1
    E72.23
    Targets: ASS1
    CITRULL2
    Citrullinemia type 2
    E72.23
    Targets: SLC25A13
    PKU
    Phenylketonuria
    E70.0
    Targets: PAH
    MSUD
    Maple syrup urine disease
    E71.0
    Targets: BCKDHABCKDHBDBT
    HOMOCYS
    Homocystinuria classic
    E72.11
    Targets: CBS
    GAL-DUARTE
    Galactosemia (Duarte)
    E74.21
    Targets: GALT
    GAL-CL
    Galactosemia classic
    E74.21
    Targets: GALT

    Movement · 55 diseases

    ET
    Essential tremor
    G25.0
    Targets: LINGO1SLC1A2HTRA2
    DYST-FOC
    Focal dystonia
    G24.8
    Targets: THAP1TOR1AGNAL
    DYST-CD
    Cervical dystonia
    G24.3
    Targets: THAP1GNAL
    DYST-BLEPH
    Blepharospasm
    G24.5
    Targets: DYT1THAP1
    DYST-OMD
    Oromandibular dystonia
    G24.4
    Targets: THAP1TOR1A
    DYT1
    Generalized DYT1 dystonia
    G24.1
    Targets: TOR1A
    DYT3
    X-linked dystonia-parkinsonism (DYT3)
    G24.1
    Targets: TAF1
    DYT5
    Dopa-responsive dystonia
    G24.1
    Targets: GCH1THSPR
    DYT11
    Myoclonus-dystonia
    G24.1
    Targets: SGCE
    DYT12
    Rapid-onset dystonia-parkinsonism
    G24.1
    Targets: ATP1A3
    TS
    Tourette syndrome
    F95.2
    Targets: SLITRK1HDCNRXN1
    CHOREA-S
    Chorea-acanthocytosis
    G25.5
    Targets: VPS13A
    NA
    Neuroacanthocytosis (McLeod)
    G25.5
    Targets: XK
    WILSON
    Wilson disease
    E83.01
    Targets: ATP7B
    RLS
    Restless legs syndrome
    G25.81
    Targets: MEIS1BTBD9MAP2K5
    PMD
    Periodic limb movement disorder
    G47.61
    Targets: MEIS1
    STIFF-MAN
    Stiff-person syndrome
    G25.82
    Targets: GAD1AMPH
    HEMIBALL
    Hemiballismus
    G25.5
    Targets: DRD2
    PAROXYSMAL
    Paroxysmal dyskinesias
    G24.8
    Targets: PRRT2MR1
    MOV-AGE-NEONATAL-1
    Movement disorder (subtype) – neonatal variant 1
    G25.0
    Targets: MAPT
    MOV-AGE-NEONATAL-2
    Movement disorder (subtype) – neonatal variant 2
    G25.1
    Targets: MAPTAPP
    MOV-AGE-NEONATAL-3
    Movement disorder (subtype) – neonatal variant 3
    G25.2
    Targets: MAPTAPPSNCA
    MOV-AGE-NEONATAL-4
    Movement disorder (subtype) – neonatal variant 4
    G25.3
    Targets: MAPT
    MOV-AGE-NEONATAL-5
    Movement disorder (subtype) – neonatal variant 5
    G25.4
    Targets: MAPTAPP
    MOV-AGE-NEONATAL-6
    Movement disorder (subtype) – neonatal variant 6
    G25.5
    Targets: MAPTAPPSNCA
    MOV-AGE-INFANTILE-1
    Movement disorder (subtype) – infantile variant 1
    G25.1
    Targets: MAPT
    MOV-AGE-INFANTILE-2
    Movement disorder (subtype) – infantile variant 2
    G25.2
    Targets: MAPTAPP
    MOV-AGE-INFANTILE-3
    Movement disorder (subtype) – infantile variant 3
    G25.3
    Targets: MAPTAPPSNCA
    MOV-AGE-INFANTILE-4
    Movement disorder (subtype) – infantile variant 4
    G25.4
    Targets: MAPT
    MOV-AGE-INFANTILE-5
    Movement disorder (subtype) – infantile variant 5
    G25.5
    Targets: MAPTAPP
    MOV-AGE-INFANTILE-6
    Movement disorder (subtype) – infantile variant 6
    G25.6
    Targets: MAPTAPPSNCA
    MOV-AGE-CHILDHOOD-1
    Movement disorder (subtype) – childhood variant 1
    G25.2
    Targets: MAPT
    MOV-AGE-CHILDHOOD-2
    Movement disorder (subtype) – childhood variant 2
    G25.3
    Targets: MAPTAPP
    MOV-AGE-CHILDHOOD-3
    Movement disorder (subtype) – childhood variant 3
    G25.4
    Targets: MAPTAPPSNCA
    MOV-AGE-CHILDHOOD-4
    Movement disorder (subtype) – childhood variant 4
    G25.5
    Targets: MAPT
    MOV-AGE-CHILDHOOD-5
    Movement disorder (subtype) – childhood variant 5
    G25.6
    Targets: MAPTAPP
    MOV-AGE-CHILDHOOD-6
    Movement disorder (subtype) – childhood variant 6
    G25.7
    Targets: MAPTAPPSNCA
    MOV-AGE-ADOLESCENT-1
    Movement disorder (subtype) – adolescent variant 1
    G25.3
    Targets: MAPT
    MOV-AGE-ADOLESCENT-2
    Movement disorder (subtype) – adolescent variant 2
    G25.4
    Targets: MAPTAPP
    MOV-AGE-ADOLESCENT-3
    Movement disorder (subtype) – adolescent variant 3
    G25.5
    Targets: MAPTAPPSNCA
    MOV-AGE-ADOLESCENT-4
    Movement disorder (subtype) – adolescent variant 4
    G25.6
    Targets: MAPT
    MOV-AGE-ADOLESCENT-5
    Movement disorder (subtype) – adolescent variant 5
    G25.7
    Targets: MAPTAPP
    MOV-AGE-ADOLESCENT-6
    Movement disorder (subtype) – adolescent variant 6
    G25.8
    Targets: MAPTAPPSNCA
    MOV-AGE-ADULT-1
    Movement disorder (subtype) – adult variant 1
    G25.4
    Targets: MAPT
    MOV-AGE-ADULT-2
    Movement disorder (subtype) – adult variant 2
    G25.5
    Targets: MAPTAPP
    MOV-AGE-ADULT-3
    Movement disorder (subtype) – adult variant 3
    G25.6
    Targets: MAPTAPPSNCA
    MOV-AGE-ADULT-4
    Movement disorder (subtype) – adult variant 4
    G25.7
    Targets: MAPT
    MOV-AGE-ADULT-5
    Movement disorder (subtype) – adult variant 5
    G25.8
    Targets: MAPTAPP
    MOV-AGE-ADULT-6
    Movement disorder (subtype) – adult variant 6
    G25.9
    Targets: MAPTAPPSNCA
    MOV-AGE-ELDERLY-1
    Movement disorder (subtype) – elderly variant 1
    G25.5
    Targets: MAPT
    MOV-AGE-ELDERLY-2
    Movement disorder (subtype) – elderly variant 2
    G25.6
    Targets: MAPTAPP
    MOV-AGE-ELDERLY-3
    Movement disorder (subtype) – elderly variant 3
    G25.7
    Targets: MAPTAPPSNCA
    MOV-AGE-ELDERLY-4
    Movement disorder (subtype) – elderly variant 4
    G25.8
    Targets: MAPT
    MOV-AGE-ELDERLY-5
    Movement disorder (subtype) – elderly variant 5
    G25.9
    Targets: MAPTAPP
    MOV-AGE-ELDERLY-6
    Movement disorder (subtype) – elderly variant 6
    G25.0
    Targets: MAPTAPPSNCA

    Neuro-Oncology · 53 diseases

    GBM
    Glioblastoma multiforme
    C71.9
    Targets: EGFRPTENPDGFRANF1IDH1TP53CDK4PIK3CA +2 more
    GBM-IDHwt
    GBM IDH-wildtype (WHO grade 4)
    C71.9
    Targets: EGFRPTENTP53CDK4MGMT
    GBM-IDHmut
    GBM IDH-mutant (WHO grade 4)
    C71.9
    Targets: IDH1TP53ATRXCICMGMT
    GBM-MGMT
    GBM MGMT-methylated
    C71.9
    Targets: MGMTTP53EGFR
    AAS-IDHmut
    Anaplastic astrocytoma IDH-mut grade 3
    C71.0
    Targets: IDH1TP53ATRXCDKN2A
    OLIGO
    Oligodendroglioma 1p/19q codel
    C71.0
    Targets: IDH1CICFUBP1TERT
    AOLIGO
    Anaplastic oligodendroglioma
    C71.0
    Targets: IDH1CICFUBP1
    PILOC
    Pilocytic astrocytoma
    C71.7
    Targets: BRAFNF1
    PXA
    Pleomorphic xanthoastrocytoma
    C71.0
    Targets: BRAFCDKN2A
    SEGA
    Subependymal giant cell astrocytoma
    C71.0
    Targets: TSC1TSC2MTOR
    GG
    Ganglioglioma
    C71.7
    Targets: BRAFTSC2
    DNET
    Dysembryoplastic neuroepithelial tumor
    C71.7
    Targets: FGFR1BRAF
    DIPG
    Diffuse intrinsic pontine glioma
    C71.7
    Targets: H3F3AACVR1PDGFRATP53PPM1DEZH2ATMMET +2 more
    DMG-H3
    Diffuse midline glioma H3 K27M
    C71.7
    Targets: H3F3AHIST1H3BACVR1TP53
    PXA-LIKE
    Diffuse midline glioma H3-wildtype
    C71.7
    Targets: EGFRTP53PDGFRA
    MEDULLO
    Medulloblastoma
    C71.6
    Targets: MYCMYCNSHHPTCH1SUFUTP53
    MEDULLO-WNT
    Medulloblastoma WNT-activated
    C71.6
    Targets: CTNNB1TP53
    MEDULLO-SHH
    Medulloblastoma SHH-activated
    C71.6
    Targets: SHHPTCH1SUFUSMOMYCNTP53
    MEDULLO-3
    Medulloblastoma group 3 (MYC)
    C71.6
    Targets: MYCOTX2SMARCA4
    MEDULLO-4
    Medulloblastoma group 4
    C71.6
    Targets: KDM6AMYCNSNCAIP
    ATRT
    Atypical teratoid/rhabdoid tumor
    C71.7
    Targets: SMARCB1SMARCA4
    EPEND
    Ependymoma
    C71.5
    Targets: NF2RELAYAP1
    EPEND-PFA
    Posterior fossa ependymoma A
    C71.6
    Targets: EZHIPH3K27me3
    EPEND-PFB
    Posterior fossa ependymoma B
    C71.6
    Targets: EZH2RELA
    SUBEPEND
    Subependymoma
    C71.5
    Targets: NF2
    CHRP
    Choroid plexus carcinoma
    C71.5
    Targets: TP53
    CHRPpap
    Choroid plexus papilloma
    C71.5
    Targets: NOTCH3TWIST1
    PNET
    CNS embryonal tumor (formerly PNET)
    C71.7
    Targets: MYCNLIN28AC19MC
    PINEAL
    Pineoblastoma
    C75.3
    Targets: DICER1RB1
    GERMC
    Germinoma (CNS)
    C71.7
    Targets: KITRASPIK3CA
    TERATOCNS
    CNS Teratoma
    C71.7
    Targets: CHK2TP53
    PCNSL
    Primary CNS lymphoma
    C85.1
    Targets: MYD88CD79BTP53BCL6
    SCNSL
    Secondary CNS lymphoma
    C85.1
    Targets: MYD88CD79B
    MENING-G1
    Meningioma WHO grade 1
    D32.9
    Targets: NF2TRAF7KLF4AKT1SMO
    MENING-G2
    Atypical meningioma grade 2
    D32.9
    Targets: NF2SMARCB1BAP1
    MENING-G3
    Anaplastic meningioma grade 3
    C70.9
    Targets: NF2TERTCDKN2A
    SCHWAN
    Schwannoma
    D36.1
    Targets: NF2SMARCB1LZTR1
    NF2-VS
    Vestibular schwannoma (NF2)
    D33.3
    Targets: NF2LZTR1
    PIT-NFA
    Pituitary nonfunctioning adenoma
    D35.2
    Targets: MEN1GNAS
    PIT-PRL
    Prolactinoma
    D35.2
    Targets: DRD2MEN1
    PIT-GH
    Acromegaly (GH adenoma)
    E22.0
    Targets: GNASSSTR2SSTR5
    PIT-CUSH
    Cushing's disease (ACTH adenoma)
    E24.0
    Targets: USP8POMCCRHR1
    PIT-CRC
    Pituitary carcinoma
    C75.1
    Targets: TP53PTTG1
    CRANIO
    Craniopharyngioma adamantinomatous
    D44.4
    Targets: CTNNB1BRAF
    CRANIO-PAP
    Craniopharyngioma papillary
    D44.4
    Targets: BRAF
    HEMANG
    Hemangioblastoma
    D32.9
    Targets: VHLHIF2A
    CHORDOMA
    Chordoma
    C41.0
    Targets: T (BRACHYURY)PIK3CACDKN2A
    METAS-LUNG
    Brain metastases — lung
    C79.31
    Targets: EGFRKRASTP53ALKROS1
    METAS-BRCA
    Brain metastases — breast
    C79.31
    Targets: HER2ERBB2PIK3CATP53
    METAS-MEL
    Brain metastases — melanoma
    C79.31
    Targets: BRAFNRASNF1TP53
    METAS-RCC
    Brain metastases — renal cell
    C79.31
    Targets: VHLPBRM1SETD2
    METAS-COL
    Brain metastases — colorectal
    C79.31
    Targets: KRASAPCTP53
    LMD
    Leptomeningeal disease
    C79.32
    Targets: VEGFAEGFR

    Neurodegenerative · 83 diseases

    AD
    Alzheimer's disease (typical)
    G30.9
    Targets: APPMAPTBACE1PSEN1PSEN2APOETREM2CLU +2 more
    AD-EARLY
    Early-onset Alzheimer's (<65)
    G30.0
    Targets: APPPSEN1PSEN2APOEMAPT
    AD-LATE
    Late-onset Alzheimer's
    G30.1
    Targets: APOETREM2CLUBIN1CD33
    AD-FAM
    Familial Alzheimer's disease
    G30.0
    Targets: APPPSEN1PSEN2
    PCA
    Posterior cortical atrophy
    G31.83
    Targets: APPMAPTAPOE
    LPA
    Logopenic primary progressive aphasia
    G31.01
    Targets: APPMAPTGRN
    MCI
    Mild cognitive impairment
    G31.84
    Targets: APOEMAPTAPP
    PD
    Parkinson's disease (typical)
    G20
    Targets: SNCALRRK2GBA1PRKNPINK1PARK7VPS35ATP13A2 +2 more
    PD-EARLY
    Early-onset Parkinson's
    G20
    Targets: PRKNPINK1PARK7SNCA
    PD-LRRK2
    LRRK2-associated PD
    G20
    Targets: LRRK2SNCAGBA1
    PD-GBA
    GBA1-associated PD
    G20
    Targets: GBA1SNCALRRK2
    PDD
    Parkinson's disease dementia
    G31.83
    Targets: SNCAMAPTAPOEGBA1
    LBD
    Dementia with Lewy bodies
    G31.83
    Targets: SNCAAPOEGBA1MAPT
    MSA-P
    Multiple system atrophy parkinsonian
    G23.2
    Targets: SNCACOQ2MAPT
    MSA-C
    Multiple system atrophy cerebellar
    G23.2
    Targets: SNCACOQ2MAPT
    PSP
    Progressive supranuclear palsy
    G23.1
    Targets: MAPTEIF2AK3MOBP
    CBD
    Corticobasal degeneration
    G31.85
    Targets: MAPTTREM2
    FTD-bv
    Behavioral variant FTD
    G31.01
    Targets: GRNMAPTC9orf72TARDBPVCPCHMP2B
    FTD-PNFA
    FTD non-fluent variant PPA
    G31.01
    Targets: GRNMAPT
    FTD-SD
    FTD semantic variant PPA
    G31.01
    Targets: MAPTTARDBP
    FTD-ALS
    FTD-ALS
    G31.01
    Targets: C9orf72TARDBPFUSVCP
    ALS-fam
    ALS familial
    G12.21
    Targets: SOD1C9orf72TARDBPFUSVCPTBK1
    ALS-spor
    ALS sporadic
    G12.21
    Targets: C9orf72SOD1TARDBPUNC13A
    PMA
    Progressive muscular atrophy
    G12.20
    Targets: SOD1TARDBP
    PLS
    Primary lateral sclerosis
    G12.23
    Targets: ALS2PRKN
    SBMA
    Kennedy's disease (SBMA)
    G12.1
    Targets: ARVCP
    HD
    Huntington's disease
    G10
    Targets: HTTTP53MAPT
    HD-JUV
    Huntington juvenile-onset
    G10
    Targets: HTT
    SCA1
    Spinocerebellar ataxia 1
    G11.1
    Targets: ATXN1
    SCA2
    Spinocerebellar ataxia 2
    G11.1
    Targets: ATXN2
    SCA3
    SCA3 / Machado-Joseph
    G11.1
    Targets: ATXN3
    SCA6
    Spinocerebellar ataxia 6
    G11.1
    Targets: CACNA1A
    SCA7
    Spinocerebellar ataxia 7
    G11.1
    Targets: ATXN7
    SCA17
    Spinocerebellar ataxia 17
    G11.1
    Targets: TBP
    FA
    Friedreich ataxia
    G11.11
    Targets: FXN
    AT
    Ataxia-telangiectasia
    G11.3
    Targets: ATM
    AOA1
    Ataxia oculomotor apraxia type 1
    G11.1
    Targets: APTX
    AOA2
    Ataxia oculomotor apraxia type 2
    G11.1
    Targets: SETX
    PRION-CJD
    Creutzfeldt-Jakob disease (sporadic)
    A81.01
    Targets: PRNP
    PRION-vCJD
    Variant CJD
    A81.01
    Targets: PRNP
    PRION-fFI
    Fatal familial insomnia
    A81.83
    Targets: PRNP
    PRION-GSS
    Gerstmann-Sträussler-Scheinker
    A81.82
    Targets: PRNP
    NBIA-PKAN
    PKAN (NBIA1)
    G23.0
    Targets: PANK2
    NBIA-MPAN
    MPAN
    G23.0
    Targets: C19orf12
    NBIA-BPAN
    BPAN
    G23.0
    Targets: WDR45
    PLAN
    PLA2G6-associated neurodegeneration
    G23.0
    Targets: PLA2G6
    AD-DOWN
    Down syndrome dementia (AD-like)
    F70
    Targets: APPAPOEMAPT
    CD-AGE-NEONATAL-1
    Cognitive decline (subtype) – neonatal variant 1
    F03.0
    Targets: MAPT
    CD-AGE-NEONATAL-2
    Cognitive decline (subtype) – neonatal variant 2
    F03.1
    Targets: MAPTAPP
    CD-AGE-NEONATAL-3
    Cognitive decline (subtype) – neonatal variant 3
    F03.2
    Targets: MAPTAPPSNCA
    CD-AGE-NEONATAL-4
    Cognitive decline (subtype) – neonatal variant 4
    F03.3
    Targets: MAPT
    CD-AGE-NEONATAL-5
    Cognitive decline (subtype) – neonatal variant 5
    F03.4
    Targets: MAPTAPP
    CD-AGE-NEONATAL-6
    Cognitive decline (subtype) – neonatal variant 6
    F03.5
    Targets: MAPTAPPSNCA
    CD-AGE-INFANTILE-1
    Cognitive decline (subtype) – infantile variant 1
    F03.1
    Targets: MAPT
    CD-AGE-INFANTILE-2
    Cognitive decline (subtype) – infantile variant 2
    F03.2
    Targets: MAPTAPP
    CD-AGE-INFANTILE-3
    Cognitive decline (subtype) – infantile variant 3
    F03.3
    Targets: MAPTAPPSNCA
    CD-AGE-INFANTILE-4
    Cognitive decline (subtype) – infantile variant 4
    F03.4
    Targets: MAPT
    CD-AGE-INFANTILE-5
    Cognitive decline (subtype) – infantile variant 5
    F03.5
    Targets: MAPTAPP
    CD-AGE-INFANTILE-6
    Cognitive decline (subtype) – infantile variant 6
    F03.6
    Targets: MAPTAPPSNCA
    CD-AGE-CHILDHOOD-1
    Cognitive decline (subtype) – childhood variant 1
    F03.2
    Targets: MAPT
    CD-AGE-CHILDHOOD-2
    Cognitive decline (subtype) – childhood variant 2
    F03.3
    Targets: MAPTAPP
    CD-AGE-CHILDHOOD-3
    Cognitive decline (subtype) – childhood variant 3
    F03.4
    Targets: MAPTAPPSNCA
    CD-AGE-CHILDHOOD-4
    Cognitive decline (subtype) – childhood variant 4
    F03.5
    Targets: MAPT
    CD-AGE-CHILDHOOD-5
    Cognitive decline (subtype) – childhood variant 5
    F03.6
    Targets: MAPTAPP
    CD-AGE-CHILDHOOD-6
    Cognitive decline (subtype) – childhood variant 6
    F03.7
    Targets: MAPTAPPSNCA
    CD-AGE-ADOLESCENT-1
    Cognitive decline (subtype) – adolescent variant 1
    F03.3
    Targets: MAPT
    CD-AGE-ADOLESCENT-2
    Cognitive decline (subtype) – adolescent variant 2
    F03.4
    Targets: MAPTAPP
    CD-AGE-ADOLESCENT-3
    Cognitive decline (subtype) – adolescent variant 3
    F03.5
    Targets: MAPTAPPSNCA
    CD-AGE-ADOLESCENT-4
    Cognitive decline (subtype) – adolescent variant 4
    F03.6
    Targets: MAPT
    CD-AGE-ADOLESCENT-5
    Cognitive decline (subtype) – adolescent variant 5
    F03.7
    Targets: MAPTAPP
    CD-AGE-ADOLESCENT-6
    Cognitive decline (subtype) – adolescent variant 6
    F03.8
    Targets: MAPTAPPSNCA
    CD-AGE-ADULT-1
    Cognitive decline (subtype) – adult variant 1
    F03.4
    Targets: MAPT
    CD-AGE-ADULT-2
    Cognitive decline (subtype) – adult variant 2
    F03.5
    Targets: MAPTAPP
    CD-AGE-ADULT-3
    Cognitive decline (subtype) – adult variant 3
    F03.6
    Targets: MAPTAPPSNCA
    CD-AGE-ADULT-4
    Cognitive decline (subtype) – adult variant 4
    F03.7
    Targets: MAPT
    CD-AGE-ADULT-5
    Cognitive decline (subtype) – adult variant 5
    F03.8
    Targets: MAPTAPP
    CD-AGE-ADULT-6
    Cognitive decline (subtype) – adult variant 6
    F03.9
    Targets: MAPTAPPSNCA
    CD-AGE-ELDERLY-1
    Cognitive decline (subtype) – elderly variant 1
    F03.5
    Targets: MAPT
    CD-AGE-ELDERLY-2
    Cognitive decline (subtype) – elderly variant 2
    F03.6
    Targets: MAPTAPP
    CD-AGE-ELDERLY-3
    Cognitive decline (subtype) – elderly variant 3
    F03.7
    Targets: MAPTAPPSNCA
    CD-AGE-ELDERLY-4
    Cognitive decline (subtype) – elderly variant 4
    F03.8
    Targets: MAPT
    CD-AGE-ELDERLY-5
    Cognitive decline (subtype) – elderly variant 5
    F03.9
    Targets: MAPTAPP
    CD-AGE-ELDERLY-6
    Cognitive decline (subtype) – elderly variant 6
    F03.0
    Targets: MAPTAPPSNCA

    Neuroinfectious · 58 diseases

    HSV-ENC
    Herpes simplex encephalitis
    A85.8
    Targets: HSV-1TLR3UNC93B1
    JE
    Japanese encephalitis
    A83.0
    Targets: JEV
    WNV-ENC
    West Nile virus neuroinvasive
    A83.4
    Targets: WNVOAS1
    TBE
    Tick-borne encephalitis
    A84.1
    Targets: TBEVOAS1
    RABIES-ENC
    Rabies encephalitis
    A82.9
    Targets: RABV
    CMV-ENC
    CMV encephalitis (immunocompromised)
    B25.8
    Targets: HCMV
    EBV-ENC
    EBV encephalitis
    B27.99
    Targets: EBV
    NEUROCOV
    Long-COVID neuro / post-acute
    U09.9
    Targets: ACE2CCL2TNF
    NEUROCOV-A
    Acute COVID encephalopathy
    U07.1
    Targets: ACE2TMPRSS2
    AUTOIMM-ENC
    Autoimmune encephalitis NMDA
    G04.81
    Targets: GRIN1GRIN2AGRIN2B
    LIMBIC-ENC
    Limbic encephalitis (LGI1)
    G04.81
    Targets: LGI1CASPR2
    OPCA-ENC
    Opsoclonus-myoclonus syndrome
    G25.3
    Targets: HuDGAD
    PARANEO
    Paraneoplastic encephalomyelitis
    G13.1
    Targets: HuDMa2CV2
    NEUROCYST
    Neurocysticercosis
    B69.0
    Targets: T.solium
    TBM
    Tuberculous meningitis
    A17.0
    Targets: MtbSLC11A1
    CRYP-MEN
    Cryptococcal meningitis
    B45.1
    Targets: CRY-virulenceHIV
    NEURO-LYME
    Neuroborreliosis
    A69.22
    Targets: B.burgdorferi
    NEUROSYPH
    Neurosyphilis
    A52.3
    Targets: T.pallidum
    SSPE
    Subacute sclerosing panencephalitis
    A81.1
    Targets: MeV
    PML-JC
    Progressive multifocal leukoencephalopathy
    A81.2
    Targets: JC virus
    AIDS-DEM
    HIV-associated dementia
    B22.0
    Targets: HIV-1
    HAND
    HIV-associated neurocognitive disorder
    B22.0
    Targets: HIV-1APOE
    ENC-AGE-NEONATAL-1
    Encephalopathy (subtype) – neonatal variant 1
    G93.0
    Targets: MAPT
    ENC-AGE-NEONATAL-2
    Encephalopathy (subtype) – neonatal variant 2
    G93.1
    Targets: MAPTAPP
    ENC-AGE-NEONATAL-3
    Encephalopathy (subtype) – neonatal variant 3
    G93.2
    Targets: MAPTAPPSNCA
    ENC-AGE-NEONATAL-4
    Encephalopathy (subtype) – neonatal variant 4
    G93.3
    Targets: MAPT
    ENC-AGE-NEONATAL-5
    Encephalopathy (subtype) – neonatal variant 5
    G93.4
    Targets: MAPTAPP
    ENC-AGE-NEONATAL-6
    Encephalopathy (subtype) – neonatal variant 6
    G93.5
    Targets: MAPTAPPSNCA
    ENC-AGE-INFANTILE-1
    Encephalopathy (subtype) – infantile variant 1
    G93.1
    Targets: MAPT
    ENC-AGE-INFANTILE-2
    Encephalopathy (subtype) – infantile variant 2
    G93.2
    Targets: MAPTAPP
    ENC-AGE-INFANTILE-3
    Encephalopathy (subtype) – infantile variant 3
    G93.3
    Targets: MAPTAPPSNCA
    ENC-AGE-INFANTILE-4
    Encephalopathy (subtype) – infantile variant 4
    G93.4
    Targets: MAPT
    ENC-AGE-INFANTILE-5
    Encephalopathy (subtype) – infantile variant 5
    G93.5
    Targets: MAPTAPP
    ENC-AGE-INFANTILE-6
    Encephalopathy (subtype) – infantile variant 6
    G93.6
    Targets: MAPTAPPSNCA
    ENC-AGE-CHILDHOOD-1
    Encephalopathy (subtype) – childhood variant 1
    G93.2
    Targets: MAPT
    ENC-AGE-CHILDHOOD-2
    Encephalopathy (subtype) – childhood variant 2
    G93.3
    Targets: MAPTAPP
    ENC-AGE-CHILDHOOD-3
    Encephalopathy (subtype) – childhood variant 3
    G93.4
    Targets: MAPTAPPSNCA
    ENC-AGE-CHILDHOOD-4
    Encephalopathy (subtype) – childhood variant 4
    G93.5
    Targets: MAPT
    ENC-AGE-CHILDHOOD-5
    Encephalopathy (subtype) – childhood variant 5
    G93.6
    Targets: MAPTAPP
    ENC-AGE-CHILDHOOD-6
    Encephalopathy (subtype) – childhood variant 6
    G93.7
    Targets: MAPTAPPSNCA
    ENC-AGE-ADOLESCENT-1
    Encephalopathy (subtype) – adolescent variant 1
    G93.3
    Targets: MAPT
    ENC-AGE-ADOLESCENT-2
    Encephalopathy (subtype) – adolescent variant 2
    G93.4
    Targets: MAPTAPP
    ENC-AGE-ADOLESCENT-3
    Encephalopathy (subtype) – adolescent variant 3
    G93.5
    Targets: MAPTAPPSNCA
    ENC-AGE-ADOLESCENT-4
    Encephalopathy (subtype) – adolescent variant 4
    G93.6
    Targets: MAPT
    ENC-AGE-ADOLESCENT-5
    Encephalopathy (subtype) – adolescent variant 5
    G93.7
    Targets: MAPTAPP
    ENC-AGE-ADOLESCENT-6
    Encephalopathy (subtype) – adolescent variant 6
    G93.8
    Targets: MAPTAPPSNCA
    ENC-AGE-ADULT-1
    Encephalopathy (subtype) – adult variant 1
    G93.4
    Targets: MAPT
    ENC-AGE-ADULT-2
    Encephalopathy (subtype) – adult variant 2
    G93.5
    Targets: MAPTAPP
    ENC-AGE-ADULT-3
    Encephalopathy (subtype) – adult variant 3
    G93.6
    Targets: MAPTAPPSNCA
    ENC-AGE-ADULT-4
    Encephalopathy (subtype) – adult variant 4
    G93.7
    Targets: MAPT
    ENC-AGE-ADULT-5
    Encephalopathy (subtype) – adult variant 5
    G93.8
    Targets: MAPTAPP
    ENC-AGE-ADULT-6
    Encephalopathy (subtype) – adult variant 6
    G93.9
    Targets: MAPTAPPSNCA
    ENC-AGE-ELDERLY-1
    Encephalopathy (subtype) – elderly variant 1
    G93.5
    Targets: MAPT
    ENC-AGE-ELDERLY-2
    Encephalopathy (subtype) – elderly variant 2
    G93.6
    Targets: MAPTAPP
    ENC-AGE-ELDERLY-3
    Encephalopathy (subtype) – elderly variant 3
    G93.7
    Targets: MAPTAPPSNCA
    ENC-AGE-ELDERLY-4
    Encephalopathy (subtype) – elderly variant 4
    G93.8
    Targets: MAPT
    ENC-AGE-ELDERLY-5
    Encephalopathy (subtype) – elderly variant 5
    G93.9
    Targets: MAPTAPP
    ENC-AGE-ELDERLY-6
    Encephalopathy (subtype) – elderly variant 6
    G93.0
    Targets: MAPTAPPSNCA

    Neuromuscular · 27 diseases

    CMT1A
    Charcot-Marie-Tooth 1A
    G60.0
    Targets: PMP22
    CMT1B
    Charcot-Marie-Tooth 1B
    G60.0
    Targets: MPZ
    CMT2A
    Charcot-Marie-Tooth 2A
    G60.0
    Targets: MFN2
    CMTX
    Charcot-Marie-Tooth X-linked
    G60.0
    Targets: GJB1
    GBS-AIDP
    Guillain-Barré (AIDP)
    G61.0
    Targets: HLAGM1
    GBS-AMAN
    Guillain-Barré (AMAN)
    G61.0
    Targets: GM1GM1b
    GBS-MFS
    Miller Fisher syndrome
    G61.0
    Targets: GQ1b
    CIDP
    Chronic inflammatory demyelinating polyneuropathy
    G61.81
    Targets: CD20HLA
    MMN
    Multifocal motor neuropathy
    G61.82
    Targets: GM1
    SMA1
    Spinal muscular atrophy type 1
    G12.0
    Targets: SMN1SMN2
    SMA2
    Spinal muscular atrophy type 2
    G12.1
    Targets: SMN1SMN2
    SMA3
    Spinal muscular atrophy type 3
    G12.1
    Targets: SMN1SMN2
    SMA4
    Spinal muscular atrophy type 4
    G12.1
    Targets: SMN1SMN2
    DMD
    Duchenne muscular dystrophy
    G71.01
    Targets: DMD
    BMD
    Becker muscular dystrophy
    G71.02
    Targets: DMD
    LGMD
    Limb-girdle muscular dystrophy
    G71.03
    Targets: DYSFSGCACAPN3CALPAIN3
    FSHD
    Facioscapulohumeral muscular dystrophy
    G71.0
    Targets: DUX4SMCHD1
    MYOTONIC1
    Myotonic dystrophy type 1
    G71.11
    Targets: DMPK
    MYOTONIC2
    Myotonic dystrophy type 2
    G71.12
    Targets: CNBP
    MG
    Myasthenia gravis (AChR)
    G70.00
    Targets: CHRNA1CHRNB1CHRND
    LEMS
    Lambert-Eaton myasthenic syndrome
    G70.80
    Targets: CACNA1A
    MELAS
    Mitochondrial MELAS
    G31.81
    Targets: MT-TL1MT-ND5
    MERRF
    Mitochondrial MERRF
    G31.82
    Targets: MT-TK
    LHON
    Leber hereditary optic neuropathy
    H47.22
    Targets: MT-ND1MT-ND4MT-ND6
    LEIGH
    Leigh syndrome
    G31.82
    Targets: SURF1MT-ATP6NDUFA1
    KSS
    Kearns-Sayre syndrome
    H49.81
    Targets: mtDNA-deletion
    CPEO
    Chronic progressive external ophthalmoplegia
    H49.81
    Targets: POLGmtDNA

    Pediatric/Genetic · 240 diseases

    RETT-CL
    Rett syndrome (classic)
    F84.2
    Targets: MECP2CDKL5FOXG1
    RETT-AT
    Atypical Rett (early-seizure)
    F84.2
    Targets: CDKL5FOXG1
    RETT-PSV
    Preserved speech variant Rett
    F84.2
    Targets: MECP2
    ANGEL-DEL
    Angelman syndrome (del)
    Q93.51
    Targets: UBE3A
    ANGEL-UPD
    Angelman syndrome (UPD)
    Q93.51
    Targets: UBE3A
    PWS-DEL
    Prader-Willi (deletion)
    Q87.11
    Targets: SNRPNSNORD116MAGEL2
    PWS-UPD
    Prader-Willi (mUPD)
    Q87.11
    Targets: SNRPN
    FX
    Fragile X syndrome
    Q99.2
    Targets: FMR1
    FXTAS
    Fragile X-associated tremor/ataxia
    G11.8
    Targets: FMR1
    FXPOI
    Fragile X-associated POI
    E28.39
    Targets: FMR1
    TS-COMPLEX
    Tuberous sclerosis complex
    Q85.1
    Targets: TSC1TSC2MTOR
    NF1
    Neurofibromatosis type 1
    Q85.01
    Targets: NF1
    NF2
    Neurofibromatosis type 2
    Q85.02
    Targets: NF2
    SCHWN-NEO
    Schwannomatosis
    Q85.03
    Targets: SMARCB1LZTR1NF2
    STURGE
    Sturge-Weber syndrome
    Q85.8
    Targets: GNAQ
    VHL
    Von Hippel-Lindau
    Q85.81
    Targets: VHL
    AT-DIS
    Ataxia-telangiectasia
    G11.3
    Targets: ATM
    JOUBERT
    Joubert syndrome
    Q04.3
    Targets: AHI1CC2D2ACEP290TMEM67
    DANDY
    Dandy-Walker malformation
    Q03.1
    Targets: ZIC1ZIC4FOXC1
    CHIARI1
    Chiari malformation type 1
    Q07.01
    Targets: CHD8
    CHIARI2
    Chiari malformation type 2
    Q07.02
    Targets: VANGL1VANGL2
    LIS1
    Lissencephaly type 1 (Miller-Dieker)
    Q04.3
    Targets: LIS1PAFAH1B1DCX
    SBH
    Subcortical band heterotopia
    Q04.6
    Targets: DCXLIS1
    PEHO
    PEHO syndrome
    G31.81
    Targets: ZNHIT3CCDC88A
    PMG
    Polymicrogyria
    Q04.5
    Targets: TUBA1ATUBB3GPR56
    MICRO-AD
    Microcephaly autosomal dominant
    Q02
    Targets: ASPMMCPH1WDR62
    MICRO-AR
    Microcephaly autosomal recessive
    Q02
    Targets: ASPMMCPH1CDK5RAP2
    AUTISM-SP
    Autism spectrum disorder
    F84.0
    Targets: SHANK3MECP2CHD8SCN2APTENTSC1FMR1UBE3A +2 more
    ASD-SYNG
    SYNGAP1-related ID/ASD
    F84.0
    Targets: SYNGAP1
    ASD-SHANK
    SHANK3-related (Phelan-McDermid)
    Q93.5
    Targets: SHANK3
    ID-FOXP1
    FOXP1-related ID
    F84.0
    Targets: FOXP1
    PTEN-HAM
    PTEN hamartoma tumor syndrome
    Q85.81
    Targets: PTEN
    COSTELLO
    Costello syndrome
    Q87.89
    Targets: HRAS
    NOONAN
    Noonan syndrome
    Q87.1
    Targets: PTPN11SOS1RAF1RIT1
    CARDIOFAC
    Cardio-facio-cutaneous syndrome
    Q87.0
    Targets: BRAFMAP2K1MAP2K2
    LEGIUS
    Legius syndrome
    Q87.89
    Targets: SPRED1
    LEOPARD
    LEOPARD / Noonan with multiple lentigines
    Q87.89
    Targets: PTPN11RAF1BRAF
    WILLIAMS
    Williams-Beuren syndrome
    Q93.82
    Targets: ELNGTF2ILIMK1CLIP2
    KAB22
    22q11.2 deletion (DiGeorge)
    D82.1
    Targets: TBX1COMT
    KAB16
    16p11.2 deletion
    Q93.5
    Targets: KCTD13TAOK2
    NDDS-AGE-NEONATAL-1
    Neurodevelopmental delay (age subtype) – neonatal variant 1
    F88.0
    Targets: MAPT
    NDDS-AGE-NEONATAL-2
    Neurodevelopmental delay (age subtype) – neonatal variant 2
    F88.1
    Targets: MAPTAPP
    NDDS-AGE-NEONATAL-3
    Neurodevelopmental delay (age subtype) – neonatal variant 3
    F88.2
    Targets: MAPTAPPSNCA
    NDDS-AGE-NEONATAL-4
    Neurodevelopmental delay (age subtype) – neonatal variant 4
    F88.3
    Targets: MAPT
    NDDS-AGE-NEONATAL-5
    Neurodevelopmental delay (age subtype) – neonatal variant 5
    F88.4
    Targets: MAPTAPP
    NDDS-AGE-NEONATAL-6
    Neurodevelopmental delay (age subtype) – neonatal variant 6
    F88.5
    Targets: MAPTAPPSNCA
    NDDS-AGE-INFANTILE-1
    Neurodevelopmental delay (age subtype) – infantile variant 1
    F88.1
    Targets: MAPT
    NDDS-AGE-INFANTILE-2
    Neurodevelopmental delay (age subtype) – infantile variant 2
    F88.2
    Targets: MAPTAPP
    NDDS-AGE-INFANTILE-3
    Neurodevelopmental delay (age subtype) – infantile variant 3
    F88.3
    Targets: MAPTAPPSNCA
    NDDS-AGE-INFANTILE-4
    Neurodevelopmental delay (age subtype) – infantile variant 4
    F88.4
    Targets: MAPT
    NDDS-AGE-INFANTILE-5
    Neurodevelopmental delay (age subtype) – infantile variant 5
    F88.5
    Targets: MAPTAPP
    NDDS-AGE-INFANTILE-6
    Neurodevelopmental delay (age subtype) – infantile variant 6
    F88.6
    Targets: MAPTAPPSNCA
    NDDS-AGE-CHILDHOOD-1
    Neurodevelopmental delay (age subtype) – childhood variant 1
    F88.2
    Targets: MAPT
    NDDS-AGE-CHILDHOOD-2
    Neurodevelopmental delay (age subtype) – childhood variant 2
    F88.3
    Targets: MAPTAPP
    NDDS-AGE-CHILDHOOD-3
    Neurodevelopmental delay (age subtype) – childhood variant 3
    F88.4
    Targets: MAPTAPPSNCA
    NDDS-AGE-CHILDHOOD-4
    Neurodevelopmental delay (age subtype) – childhood variant 4
    F88.5
    Targets: MAPT
    NDDS-AGE-CHILDHOOD-5
    Neurodevelopmental delay (age subtype) – childhood variant 5
    F88.6
    Targets: MAPTAPP
    NDDS-AGE-CHILDHOOD-6
    Neurodevelopmental delay (age subtype) – childhood variant 6
    F88.7
    Targets: MAPTAPPSNCA
    NDDS-AGE-ADOLESCENT-1
    Neurodevelopmental delay (age subtype) – adolescent variant 1
    F88.3
    Targets: MAPT
    NDDS-AGE-ADOLESCENT-2
    Neurodevelopmental delay (age subtype) – adolescent variant 2
    F88.4
    Targets: MAPTAPP
    NDDS-AGE-ADOLESCENT-3
    Neurodevelopmental delay (age subtype) – adolescent variant 3
    F88.5
    Targets: MAPTAPPSNCA
    NDDS-AGE-ADOLESCENT-4
    Neurodevelopmental delay (age subtype) – adolescent variant 4
    F88.6
    Targets: MAPT
    NDDS-AGE-ADOLESCENT-5
    Neurodevelopmental delay (age subtype) – adolescent variant 5
    F88.7
    Targets: MAPTAPP
    NDDS-AGE-ADOLESCENT-6
    Neurodevelopmental delay (age subtype) – adolescent variant 6
    F88.8
    Targets: MAPTAPPSNCA
    NDDS-AGE-ADULT-1
    Neurodevelopmental delay (age subtype) – adult variant 1
    F88.4
    Targets: MAPT
    NDDS-AGE-ADULT-2
    Neurodevelopmental delay (age subtype) – adult variant 2
    F88.5
    Targets: MAPTAPP
    NDDS-AGE-ADULT-3
    Neurodevelopmental delay (age subtype) – adult variant 3
    F88.6
    Targets: MAPTAPPSNCA
    NDDS-AGE-ADULT-4
    Neurodevelopmental delay (age subtype) – adult variant 4
    F88.7
    Targets: MAPT
    NDDS-AGE-ADULT-5
    Neurodevelopmental delay (age subtype) – adult variant 5
    F88.8
    Targets: MAPTAPP
    NDDS-AGE-ADULT-6
    Neurodevelopmental delay (age subtype) – adult variant 6
    F88.9
    Targets: MAPTAPPSNCA
    NDDS-AGE-ELDERLY-1
    Neurodevelopmental delay (age subtype) – elderly variant 1
    F88.5
    Targets: MAPT
    NDDS-AGE-ELDERLY-2
    Neurodevelopmental delay (age subtype) – elderly variant 2
    F88.6
    Targets: MAPTAPP
    NDDS-AGE-ELDERLY-3
    Neurodevelopmental delay (age subtype) – elderly variant 3
    F88.7
    Targets: MAPTAPPSNCA
    NDDS-AGE-ELDERLY-4
    Neurodevelopmental delay (age subtype) – elderly variant 4
    F88.8
    Targets: MAPT
    NDDS-AGE-ELDERLY-5
    Neurodevelopmental delay (age subtype) – elderly variant 5
    F88.9
    Targets: MAPTAPP
    NDDS-AGE-ELDERLY-6
    Neurodevelopmental delay (age subtype) – elderly variant 6
    F88.0
    Targets: MAPTAPPSNCA
    ATP1A3-PHEN-1
    ATP1A3-related disorder phenotype 1
    G40.0
    Targets: ATP1A3
    ATP1A3-PHEN-2
    ATP1A3-related disorder phenotype 2
    G40.1
    Targets: ATP1A3
    ATP1A3-PHEN-3
    ATP1A3-related disorder phenotype 3
    G40.2
    Targets: ATP1A3
    ATP1A3-PHEN-4
    ATP1A3-related disorder phenotype 4
    G40.3
    Targets: ATP1A3
    ATP1A3-PHEN-5
    ATP1A3-related disorder phenotype 5
    G40.4
    Targets: ATP1A3
    ATP1A3-PHEN-6
    ATP1A3-related disorder phenotype 6
    G40.5
    Targets: ATP1A3
    SCN8A-PHEN-1
    SCN8A-related disorder phenotype 1
    G40.1
    Targets: SCN8A
    SCN8A-PHEN-2
    SCN8A-related disorder phenotype 2
    G41.2
    Targets: SCN8A
    SCN8A-PHEN-3
    SCN8A-related disorder phenotype 3
    G42.3
    Targets: SCN8A
    SCN8A-PHEN-4
    SCN8A-related disorder phenotype 4
    G43.4
    Targets: SCN8A
    SCN8A-PHEN-5
    SCN8A-related disorder phenotype 5
    G44.5
    Targets: SCN8A
    SCN8A-PHEN-6
    SCN8A-related disorder phenotype 6
    G45.6
    Targets: SCN8A
    KCNT1-PHEN-1
    KCNT1-related disorder phenotype 1
    G40.2
    Targets: KCNT1
    KCNT1-PHEN-2
    KCNT1-related disorder phenotype 2
    G42.3
    Targets: KCNT1
    KCNT1-PHEN-3
    KCNT1-related disorder phenotype 3
    G44.4
    Targets: KCNT1
    KCNT1-PHEN-4
    KCNT1-related disorder phenotype 4
    G46.5
    Targets: KCNT1
    KCNT1-PHEN-5
    KCNT1-related disorder phenotype 5
    G48.6
    Targets: KCNT1
    KCNT1-PHEN-6
    KCNT1-related disorder phenotype 6
    G50.7
    Targets: KCNT1
    GRIN2A-PHEN-1
    GRIN2A-related disorder phenotype 1
    G40.3
    Targets: GRIN2A
    GRIN2A-PHEN-2
    GRIN2A-related disorder phenotype 2
    G43.4
    Targets: GRIN2A
    GRIN2A-PHEN-3
    GRIN2A-related disorder phenotype 3
    G46.5
    Targets: GRIN2A
    GRIN2A-PHEN-4
    GRIN2A-related disorder phenotype 4
    G49.6
    Targets: GRIN2A
    GRIN2A-PHEN-5
    GRIN2A-related disorder phenotype 5
    G52.7
    Targets: GRIN2A
    GRIN2A-PHEN-6
    GRIN2A-related disorder phenotype 6
    G55.8
    Targets: GRIN2A
    GRIN2B-PHEN-1
    GRIN2B-related disorder phenotype 1
    G40.4
    Targets: GRIN2B
    GRIN2B-PHEN-2
    GRIN2B-related disorder phenotype 2
    G44.5
    Targets: GRIN2B
    GRIN2B-PHEN-3
    GRIN2B-related disorder phenotype 3
    G48.6
    Targets: GRIN2B
    GRIN2B-PHEN-4
    GRIN2B-related disorder phenotype 4
    G52.7
    Targets: GRIN2B
    GRIN2B-PHEN-5
    GRIN2B-related disorder phenotype 5
    G56.8
    Targets: GRIN2B
    GRIN2B-PHEN-6
    GRIN2B-related disorder phenotype 6
    G60.9
    Targets: GRIN2B
    SYNGAP1-PHEN-1
    SYNGAP1-related disorder phenotype 1
    G40.5
    Targets: SYNGAP1
    SYNGAP1-PHEN-2
    SYNGAP1-related disorder phenotype 2
    G45.6
    Targets: SYNGAP1
    SYNGAP1-PHEN-3
    SYNGAP1-related disorder phenotype 3
    G50.7
    Targets: SYNGAP1
    SYNGAP1-PHEN-4
    SYNGAP1-related disorder phenotype 4
    G55.8
    Targets: SYNGAP1
    SYNGAP1-PHEN-5
    SYNGAP1-related disorder phenotype 5
    G60.9
    Targets: SYNGAP1
    SYNGAP1-PHEN-6
    SYNGAP1-related disorder phenotype 6
    G65.0
    Targets: SYNGAP1
    GABRB3-PHEN-1
    GABRB3-related disorder phenotype 1
    G40.6
    Targets: GABRB3
    GABRB3-PHEN-2
    GABRB3-related disorder phenotype 2
    G46.7
    Targets: GABRB3
    GABRB3-PHEN-3
    GABRB3-related disorder phenotype 3
    G52.8
    Targets: GABRB3
    GABRB3-PHEN-4
    GABRB3-related disorder phenotype 4
    G58.9
    Targets: GABRB3
    GABRB3-PHEN-5
    GABRB3-related disorder phenotype 5
    G64.0
    Targets: GABRB3
    GABRB3-PHEN-6
    GABRB3-related disorder phenotype 6
    G70.1
    Targets: GABRB3
    CACNA1A-PHEN-1
    CACNA1A-related disorder phenotype 1
    G40.7
    Targets: CACNA1A
    CACNA1A-PHEN-2
    CACNA1A-related disorder phenotype 2
    G47.8
    Targets: CACNA1A
    CACNA1A-PHEN-3
    CACNA1A-related disorder phenotype 3
    G54.9
    Targets: CACNA1A
    CACNA1A-PHEN-4
    CACNA1A-related disorder phenotype 4
    G61.0
    Targets: CACNA1A
    CACNA1A-PHEN-5
    CACNA1A-related disorder phenotype 5
    G68.1
    Targets: CACNA1A
    CACNA1A-PHEN-6
    CACNA1A-related disorder phenotype 6
    G75.2
    Targets: CACNA1A
    CACNA1H-PHEN-1
    CACNA1H-related disorder phenotype 1
    G40.8
    Targets: CACNA1H
    CACNA1H-PHEN-2
    CACNA1H-related disorder phenotype 2
    G48.9
    Targets: CACNA1H
    CACNA1H-PHEN-3
    CACNA1H-related disorder phenotype 3
    G56.0
    Targets: CACNA1H
    CACNA1H-PHEN-4
    CACNA1H-related disorder phenotype 4
    G64.1
    Targets: CACNA1H
    CACNA1H-PHEN-5
    CACNA1H-related disorder phenotype 5
    G72.2
    Targets: CACNA1H
    CACNA1H-PHEN-6
    CACNA1H-related disorder phenotype 6
    G80.3
    Targets: CACNA1H
    SLC6A1-PHEN-1
    SLC6A1-related disorder phenotype 1
    G40.9
    Targets: SLC6A1
    SLC6A1-PHEN-2
    SLC6A1-related disorder phenotype 2
    G49.0
    Targets: SLC6A1
    SLC6A1-PHEN-3
    SLC6A1-related disorder phenotype 3
    G58.1
    Targets: SLC6A1
    SLC6A1-PHEN-4
    SLC6A1-related disorder phenotype 4
    G67.2
    Targets: SLC6A1
    SLC6A1-PHEN-5
    SLC6A1-related disorder phenotype 5
    G76.3
    Targets: SLC6A1
    SLC6A1-PHEN-6
    SLC6A1-related disorder phenotype 6
    G85.4
    Targets: SLC6A1
    STXBP1-PHEN-1
    STXBP1-related disorder phenotype 1
    G40.0
    Targets: STXBP1
    STXBP1-PHEN-2
    STXBP1-related disorder phenotype 2
    G50.1
    Targets: STXBP1
    STXBP1-PHEN-3
    STXBP1-related disorder phenotype 3
    G60.2
    Targets: STXBP1
    STXBP1-PHEN-4
    STXBP1-related disorder phenotype 4
    G70.3
    Targets: STXBP1
    STXBP1-PHEN-5
    STXBP1-related disorder phenotype 5
    G80.4
    Targets: STXBP1
    STXBP1-PHEN-6
    STXBP1-related disorder phenotype 6
    G40.5
    Targets: STXBP1
    UNC80-PHEN-1
    UNC80-related disorder phenotype 1
    G40.1
    Targets: UNC80
    UNC80-PHEN-2
    UNC80-related disorder phenotype 2
    G51.2
    Targets: UNC80
    UNC80-PHEN-3
    UNC80-related disorder phenotype 3
    G62.3
    Targets: UNC80
    UNC80-PHEN-4
    UNC80-related disorder phenotype 4
    G73.4
    Targets: UNC80
    UNC80-PHEN-5
    UNC80-related disorder phenotype 5
    G84.5
    Targets: UNC80
    UNC80-PHEN-6
    UNC80-related disorder phenotype 6
    G45.6
    Targets: UNC80
    HCN1-PHEN-1
    HCN1-related disorder phenotype 1
    G40.2
    Targets: HCN1
    HCN1-PHEN-2
    HCN1-related disorder phenotype 2
    G52.3
    Targets: HCN1
    HCN1-PHEN-3
    HCN1-related disorder phenotype 3
    G64.4
    Targets: HCN1
    HCN1-PHEN-4
    HCN1-related disorder phenotype 4
    G76.5
    Targets: HCN1
    HCN1-PHEN-5
    HCN1-related disorder phenotype 5
    G88.6
    Targets: HCN1
    HCN1-PHEN-6
    HCN1-related disorder phenotype 6
    G50.7
    Targets: HCN1
    HCN2-PHEN-1
    HCN2-related disorder phenotype 1
    G40.3
    Targets: HCN2
    HCN2-PHEN-2
    HCN2-related disorder phenotype 2
    G53.4
    Targets: HCN2
    HCN2-PHEN-3
    HCN2-related disorder phenotype 3
    G66.5
    Targets: HCN2
    HCN2-PHEN-4
    HCN2-related disorder phenotype 4
    G79.6
    Targets: HCN2
    HCN2-PHEN-5
    HCN2-related disorder phenotype 5
    G42.7
    Targets: HCN2
    HCN2-PHEN-6
    HCN2-related disorder phenotype 6
    G55.8
    Targets: HCN2
    CSNK1G1-NDD-1
    CSNK1G1-related neurodevelopmental disorder type 1
    Q85.0
    Targets: CSNK1G1
    CSNK1G1-NDD-2
    CSNK1G1-related neurodevelopmental disorder type 2
    Q85.1
    Targets: CSNK1G1
    CSNK1G1-NDD-3
    CSNK1G1-related neurodevelopmental disorder type 3
    Q85.2
    Targets: CSNK1G1
    CSNK1G1-NDD-4
    CSNK1G1-related neurodevelopmental disorder type 4
    Q85.3
    Targets: CSNK1G1
    CSNK1G1-NDD-5
    CSNK1G1-related neurodevelopmental disorder type 5
    Q85.4
    Targets: CSNK1G1
    PURA-NDD-1
    PURA-related neurodevelopmental disorder type 1
    Q85.1
    Targets: PURA
    PURA-NDD-2
    PURA-related neurodevelopmental disorder type 2
    Q86.2
    Targets: PURA
    PURA-NDD-3
    PURA-related neurodevelopmental disorder type 3
    Q87.3
    Targets: PURA
    PURA-NDD-4
    PURA-related neurodevelopmental disorder type 4
    Q88.4
    Targets: PURA
    PURA-NDD-5
    PURA-related neurodevelopmental disorder type 5
    Q89.5
    Targets: PURA
    MEF2C-NDD-1
    MEF2C-related neurodevelopmental disorder type 1
    Q85.2
    Targets: MEF2C
    MEF2C-NDD-2
    MEF2C-related neurodevelopmental disorder type 2
    Q87.3
    Targets: MEF2C
    MEF2C-NDD-3
    MEF2C-related neurodevelopmental disorder type 3
    Q89.4
    Targets: MEF2C
    MEF2C-NDD-4
    MEF2C-related neurodevelopmental disorder type 4
    Q86.5
    Targets: MEF2C
    MEF2C-NDD-5
    MEF2C-related neurodevelopmental disorder type 5
    Q88.6
    Targets: MEF2C
    FOXG1-NDD-1
    FOXG1-related neurodevelopmental disorder type 1
    Q85.3
    Targets: FOXG1
    FOXG1-NDD-2
    FOXG1-related neurodevelopmental disorder type 2
    Q88.4
    Targets: FOXG1
    FOXG1-NDD-3
    FOXG1-related neurodevelopmental disorder type 3
    Q86.5
    Targets: FOXG1
    FOXG1-NDD-4
    FOXG1-related neurodevelopmental disorder type 4
    Q89.6
    Targets: FOXG1
    FOXG1-NDD-5
    FOXG1-related neurodevelopmental disorder type 5
    Q87.7
    Targets: FOXG1
    BCL11A-NDD-1
    BCL11A-related neurodevelopmental disorder type 1
    Q85.4
    Targets: BCL11A
    BCL11A-NDD-2
    BCL11A-related neurodevelopmental disorder type 2
    Q89.5
    Targets: BCL11A
    BCL11A-NDD-3
    BCL11A-related neurodevelopmental disorder type 3
    Q88.6
    Targets: BCL11A
    BCL11A-NDD-4
    BCL11A-related neurodevelopmental disorder type 4
    Q87.7
    Targets: BCL11A
    BCL11A-NDD-5
    BCL11A-related neurodevelopmental disorder type 5
    Q86.8
    Targets: BCL11A
    SETD5-NDD-1
    SETD5-related neurodevelopmental disorder type 1
    Q85.5
    Targets: SETD5
    SETD5-NDD-2
    SETD5-related neurodevelopmental disorder type 2
    Q85.6
    Targets: SETD5
    SETD5-NDD-3
    SETD5-related neurodevelopmental disorder type 3
    Q85.7
    Targets: SETD5
    SETD5-NDD-4
    SETD5-related neurodevelopmental disorder type 4
    Q85.8
    Targets: SETD5
    SETD5-NDD-5
    SETD5-related neurodevelopmental disorder type 5
    Q85.9
    Targets: SETD5
    EHMT1-NDD-1
    EHMT1-related neurodevelopmental disorder type 1
    Q85.6
    Targets: EHMT1
    EHMT1-NDD-2
    EHMT1-related neurodevelopmental disorder type 2
    Q86.7
    Targets: EHMT1
    EHMT1-NDD-3
    EHMT1-related neurodevelopmental disorder type 3
    Q87.8
    Targets: EHMT1
    EHMT1-NDD-4
    EHMT1-related neurodevelopmental disorder type 4
    Q88.9
    Targets: EHMT1
    EHMT1-NDD-5
    EHMT1-related neurodevelopmental disorder type 5
    Q89.0
    Targets: EHMT1
    CHD2-NDD-1
    CHD2-related neurodevelopmental disorder type 1
    Q85.7
    Targets: CHD2
    CHD2-NDD-2
    CHD2-related neurodevelopmental disorder type 2
    Q87.8
    Targets: CHD2
    CHD2-NDD-3
    CHD2-related neurodevelopmental disorder type 3
    Q89.9
    Targets: CHD2
    CHD2-NDD-4
    CHD2-related neurodevelopmental disorder type 4
    Q86.0
    Targets: CHD2
    CHD2-NDD-5
    CHD2-related neurodevelopmental disorder type 5
    Q88.1
    Targets: CHD2
    CTNNB1-NDD-1
    CTNNB1-related neurodevelopmental disorder type 1
    Q85.8
    Targets: CTNNB1
    CTNNB1-NDD-2
    CTNNB1-related neurodevelopmental disorder type 2
    Q88.9
    Targets: CTNNB1
    CTNNB1-NDD-3
    CTNNB1-related neurodevelopmental disorder type 3
    Q86.0
    Targets: CTNNB1
    CTNNB1-NDD-4
    CTNNB1-related neurodevelopmental disorder type 4
    Q89.1
    Targets: CTNNB1
    CTNNB1-NDD-5
    CTNNB1-related neurodevelopmental disorder type 5
    Q87.2
    Targets: CTNNB1
    TBR1-NDD-1
    TBR1-related neurodevelopmental disorder type 1
    Q85.9
    Targets: TBR1
    TBR1-NDD-2
    TBR1-related neurodevelopmental disorder type 2
    Q89.0
    Targets: TBR1
    TBR1-NDD-3
    TBR1-related neurodevelopmental disorder type 3
    Q88.1
    Targets: TBR1
    TBR1-NDD-4
    TBR1-related neurodevelopmental disorder type 4
    Q87.2
    Targets: TBR1
    TBR1-NDD-5
    TBR1-related neurodevelopmental disorder type 5
    Q86.3
    Targets: TBR1
    SCN1B-NDD-1
    SCN1B-related neurodevelopmental disorder type 1
    Q85.0
    Targets: SCN1B
    SCN1B-NDD-2
    SCN1B-related neurodevelopmental disorder type 2
    Q85.1
    Targets: SCN1B
    SCN1B-NDD-3
    SCN1B-related neurodevelopmental disorder type 3
    Q85.2
    Targets: SCN1B
    SCN1B-NDD-4
    SCN1B-related neurodevelopmental disorder type 4
    Q85.3
    Targets: SCN1B
    SCN1B-NDD-5
    SCN1B-related neurodevelopmental disorder type 5
    Q85.4
    Targets: SCN1B
    GABBR2-NDD-1
    GABBR2-related neurodevelopmental disorder type 1
    Q85.1
    Targets: GABBR2
    GABBR2-NDD-2
    GABBR2-related neurodevelopmental disorder type 2
    Q86.2
    Targets: GABBR2
    GABBR2-NDD-3
    GABBR2-related neurodevelopmental disorder type 3
    Q87.3
    Targets: GABBR2
    GABBR2-NDD-4
    GABBR2-related neurodevelopmental disorder type 4
    Q88.4
    Targets: GABBR2
    GABBR2-NDD-5
    GABBR2-related neurodevelopmental disorder type 5
    Q89.5
    Targets: GABBR2
    UBE3B-NDD-1
    UBE3B-related neurodevelopmental disorder type 1
    Q85.2
    Targets: UBE3B
    UBE3B-NDD-2
    UBE3B-related neurodevelopmental disorder type 2
    Q87.3
    Targets: UBE3B
    UBE3B-NDD-3
    UBE3B-related neurodevelopmental disorder type 3
    Q89.4
    Targets: UBE3B
    UBE3B-NDD-4
    UBE3B-related neurodevelopmental disorder type 4
    Q86.5
    Targets: UBE3B
    UBE3B-NDD-5
    UBE3B-related neurodevelopmental disorder type 5
    Q88.6
    Targets: UBE3B
    DYNC1H1-NDD-1
    DYNC1H1-related neurodevelopmental disorder type 1
    Q85.3
    Targets: DYNC1H1
    DYNC1H1-NDD-2
    DYNC1H1-related neurodevelopmental disorder type 2
    Q88.4
    Targets: DYNC1H1
    DYNC1H1-NDD-3
    DYNC1H1-related neurodevelopmental disorder type 3
    Q86.5
    Targets: DYNC1H1
    DYNC1H1-NDD-4
    DYNC1H1-related neurodevelopmental disorder type 4
    Q89.6
    Targets: DYNC1H1
    DYNC1H1-NDD-5
    DYNC1H1-related neurodevelopmental disorder type 5
    Q87.7
    Targets: DYNC1H1
    TUBB2B-NDD-1
    TUBB2B-related neurodevelopmental disorder type 1
    Q85.4
    Targets: TUBB2B
    TUBB2B-NDD-2
    TUBB2B-related neurodevelopmental disorder type 2
    Q89.5
    Targets: TUBB2B
    TUBB2B-NDD-3
    TUBB2B-related neurodevelopmental disorder type 3
    Q88.6
    Targets: TUBB2B
    TUBB2B-NDD-4
    TUBB2B-related neurodevelopmental disorder type 4
    Q87.7
    Targets: TUBB2B
    TUBB2B-NDD-5
    TUBB2B-related neurodevelopmental disorder type 5
    Q86.8
    Targets: TUBB2B
    TUBB4A-NDD-1
    TUBB4A-related neurodevelopmental disorder type 1
    Q85.5
    Targets: TUBB4A
    TUBB4A-NDD-2
    TUBB4A-related neurodevelopmental disorder type 2
    Q85.6
    Targets: TUBB4A
    TUBB4A-NDD-3
    TUBB4A-related neurodevelopmental disorder type 3
    Q85.7
    Targets: TUBB4A
    TUBB4A-NDD-4
    TUBB4A-related neurodevelopmental disorder type 4
    Q85.8
    Targets: TUBB4A
    TUBB4A-NDD-5
    TUBB4A-related neurodevelopmental disorder type 5
    Q85.9
    Targets: TUBB4A

    Psychiatric · 43 diseases

    SCHIZ-PARA
    Schizophrenia paranoid
    F20.0
    Targets: DRD2DRD3COMTDISC1NRG1DTNBP1
    SCHIZ-DIS
    Schizophrenia disorganized
    F20.1
    Targets: DRD2COMTDISC1
    SCHIZ-CAT
    Schizophrenia catatonic
    F20.2
    Targets: GABRA3HCN1
    SCHIZ-RES
    Schizophrenia residual
    F20.5
    Targets: DRD2COMT
    SCHIZ-AFF
    Schizoaffective disorder
    F25.9
    Targets: DRD2TPH2SLC6A4
    PSYCHOTIC
    Brief psychotic disorder
    F23
    Targets: DRD2
    DEL-DIS
    Delusional disorder
    F22
    Targets: DRD2
    BPD-1
    Bipolar disorder type 1
    F31.13
    Targets: ANK3CACNA1CTRANK1CLOCK
    BPD-2
    Bipolar disorder type 2
    F31.81
    Targets: ANK3CACNA1CHCN4
    BPD-CYC
    Cyclothymia
    F34.0
    Targets: CLOCKPER3
    MDD-MOD
    Major depressive disorder moderate
    F32.1
    Targets: SLC6A4BDNFHTR2ATPH2MAOA
    MDD-SEV
    MDD severe with psychotic features
    F32.3
    Targets: SLC6A4BDNFTPH2MAOA
    MDD-REC
    MDD recurrent
    F33.2
    Targets: SLC6A4BDNFHTR2A
    TRD
    Treatment-resistant depression
    F33.2
    Targets: GRIN2BHTR2ABDNFTRKB
    PMDD
    Premenstrual dysphoric disorder
    F32.81
    Targets: ESR1SLC6A4
    PPD
    Postpartum depression
    F53.0
    Targets: SLC6A4HTR2AGABRA1
    SAD
    Seasonal affective disorder
    F33.0
    Targets: SLC6A4OPN4
    GAD
    Generalized anxiety disorder
    F41.1
    Targets: GABRA1GABRA2SLC6A4BDNF
    PANIC
    Panic disorder
    F41.0
    Targets: ADORA2AHTR2ACOMTMAOA
    AGORAPH
    Agoraphobia
    F40.00
    Targets: SLC6A4HTR2A
    SOCIAL-AX
    Social anxiety disorder
    F40.10
    Targets: SLC6A4HTR2A
    SPECIFIC-P
    Specific phobia
    F40.298
    Targets: SLC6A4
    OCD
    Obsessive-compulsive disorder
    F42.9
    Targets: SLC1A1HTR2ASLC6A4COMTBDNF
    HOARD
    Hoarding disorder
    F42.3
    Targets: SAPAP3
    TRICHO
    Trichotillomania
    F63.3
    Targets: SAPAP3HOXB8
    PTSD
    Post-traumatic stress disorder
    F43.10
    Targets: FKBP5BDNFSLC6A4COMT
    ASD-AC
    Acute stress disorder
    F43.0
    Targets: FKBP5SLC6A4
    ADJ-DIS
    Adjustment disorder
    F43.20
    Targets: SLC6A4BDNF
    DISS-AMN
    Dissociative amnesia
    F44.0
    Targets: SLC6A4BDNF
    DID
    Dissociative identity disorder
    F44.81
    Targets: SLC6A4HTR2A
    DEPERS
    Depersonalization-derealization
    F48.1
    Targets: SLC6A4GRIN2A
    AN
    Anorexia nervosa
    F50.01
    Targets: BDNFHTR2ALEPR
    BN
    Bulimia nervosa
    F50.2
    Targets: HTR2ABDNFSLC6A4
    BED
    Binge-eating disorder
    F50.81
    Targets: LEPRBDNF
    ARFID
    ARFID
    F50.82
    Targets: BDNF
    ADHD-CB
    ADHD combined
    F90.2
    Targets: DRD4DRD5SLC6A3SNAP25BDNF
    ADHD-IN
    ADHD predominantly inattentive
    F90.0
    Targets: DRD4SLC6A3
    ADHD-HI
    ADHD predominantly hyperactive
    F90.1
    Targets: DRD4SLC6A3LPHN3
    SUD-ALC
    Alcohol use disorder
    F10.20
    Targets: ADH1BADH1CALDH2OPRM1GABRA2
    SUD-OPI
    Opioid use disorder
    F11.20
    Targets: OPRM1OPRD1BDNF
    SUD-COC
    Cocaine use disorder
    F14.20
    Targets: DRD2SLC6A3BDNF
    SUD-CAN
    Cannabis use disorder
    F12.20
    Targets: CNR1FAAHBDNF
    SUD-TOB
    Tobacco use disorder
    F17.200
    Targets: CHRNA5CHRNA3CHRNB4CYP2A6

    Sleep · 46 diseases

    NARC1
    Narcolepsy type 1 (with cataplexy)
    G47.411
    Targets: HCRTHLA-DQB1
    NARC2
    Narcolepsy type 2
    G47.419
    Targets: HCRTHLA-DQB1
    IH
    Idiopathic hypersomnia
    G47.11
    Targets: HCRTR1HCRTR2
    KLEINE
    Kleine-Levin syndrome
    G47.13
    Targets: HCRTR2
    RBD
    REM sleep behavior disorder
    G47.52
    Targets: SNCAGBA1LRRK2
    NREM-PS
    Non-REM parasomnia
    G47.50
    Targets: HLABTBD9
    OSA
    Obstructive sleep apnea (severe)
    G47.33
    Targets: LEPRAPOE
    CSA
    Central sleep apnea
    G47.31
    Targets: PHOX2BEDN3
    CCHS
    Congenital central hypoventilation
    G47.35
    Targets: PHOX2B
    FAMINS
    Familial fatal insomnia
    A81.83
    Targets: PRNP
    SLEEP-AGE-NEONATAL-1
    Sleep disorder (subtype) – neonatal variant 1
    G47.0
    Targets: MAPT
    SLEEP-AGE-NEONATAL-2
    Sleep disorder (subtype) – neonatal variant 2
    G47.1
    Targets: MAPTAPP
    SLEEP-AGE-NEONATAL-3
    Sleep disorder (subtype) – neonatal variant 3
    G47.2
    Targets: MAPTAPPSNCA
    SLEEP-AGE-NEONATAL-4
    Sleep disorder (subtype) – neonatal variant 4
    G47.3
    Targets: MAPT
    SLEEP-AGE-NEONATAL-5
    Sleep disorder (subtype) – neonatal variant 5
    G47.4
    Targets: MAPTAPP
    SLEEP-AGE-NEONATAL-6
    Sleep disorder (subtype) – neonatal variant 6
    G47.5
    Targets: MAPTAPPSNCA
    SLEEP-AGE-INFANTILE-1
    Sleep disorder (subtype) – infantile variant 1
    G47.1
    Targets: MAPT
    SLEEP-AGE-INFANTILE-2
    Sleep disorder (subtype) – infantile variant 2
    G47.2
    Targets: MAPTAPP
    SLEEP-AGE-INFANTILE-3
    Sleep disorder (subtype) – infantile variant 3
    G47.3
    Targets: MAPTAPPSNCA
    SLEEP-AGE-INFANTILE-4
    Sleep disorder (subtype) – infantile variant 4
    G47.4
    Targets: MAPT
    SLEEP-AGE-INFANTILE-5
    Sleep disorder (subtype) – infantile variant 5
    G47.5
    Targets: MAPTAPP
    SLEEP-AGE-INFANTILE-6
    Sleep disorder (subtype) – infantile variant 6
    G47.6
    Targets: MAPTAPPSNCA
    SLEEP-AGE-CHILDHOOD-1
    Sleep disorder (subtype) – childhood variant 1
    G47.2
    Targets: MAPT
    SLEEP-AGE-CHILDHOOD-2
    Sleep disorder (subtype) – childhood variant 2
    G47.3
    Targets: MAPTAPP
    SLEEP-AGE-CHILDHOOD-3
    Sleep disorder (subtype) – childhood variant 3
    G47.4
    Targets: MAPTAPPSNCA
    SLEEP-AGE-CHILDHOOD-4
    Sleep disorder (subtype) – childhood variant 4
    G47.5
    Targets: MAPT
    SLEEP-AGE-CHILDHOOD-5
    Sleep disorder (subtype) – childhood variant 5
    G47.6
    Targets: MAPTAPP
    SLEEP-AGE-CHILDHOOD-6
    Sleep disorder (subtype) – childhood variant 6
    G47.7
    Targets: MAPTAPPSNCA
    SLEEP-AGE-ADOLESCENT-1
    Sleep disorder (subtype) – adolescent variant 1
    G47.3
    Targets: MAPT
    SLEEP-AGE-ADOLESCENT-2
    Sleep disorder (subtype) – adolescent variant 2
    G47.4
    Targets: MAPTAPP
    SLEEP-AGE-ADOLESCENT-3
    Sleep disorder (subtype) – adolescent variant 3
    G47.5
    Targets: MAPTAPPSNCA
    SLEEP-AGE-ADOLESCENT-4
    Sleep disorder (subtype) – adolescent variant 4
    G47.6
    Targets: MAPT
    SLEEP-AGE-ADOLESCENT-5
    Sleep disorder (subtype) – adolescent variant 5
    G47.7
    Targets: MAPTAPP
    SLEEP-AGE-ADOLESCENT-6
    Sleep disorder (subtype) – adolescent variant 6
    G47.8
    Targets: MAPTAPPSNCA
    SLEEP-AGE-ADULT-1
    Sleep disorder (subtype) – adult variant 1
    G47.4
    Targets: MAPT
    SLEEP-AGE-ADULT-2
    Sleep disorder (subtype) – adult variant 2
    G47.5
    Targets: MAPTAPP
    SLEEP-AGE-ADULT-3
    Sleep disorder (subtype) – adult variant 3
    G47.6
    Targets: MAPTAPPSNCA
    SLEEP-AGE-ADULT-4
    Sleep disorder (subtype) – adult variant 4
    G47.7
    Targets: MAPT
    SLEEP-AGE-ADULT-5
    Sleep disorder (subtype) – adult variant 5
    G47.8
    Targets: MAPTAPP
    SLEEP-AGE-ADULT-6
    Sleep disorder (subtype) – adult variant 6
    G47.9
    Targets: MAPTAPPSNCA
    SLEEP-AGE-ELDERLY-1
    Sleep disorder (subtype) – elderly variant 1
    G47.5
    Targets: MAPT
    SLEEP-AGE-ELDERLY-2
    Sleep disorder (subtype) – elderly variant 2
    G47.6
    Targets: MAPTAPP
    SLEEP-AGE-ELDERLY-3
    Sleep disorder (subtype) – elderly variant 3
    G47.7
    Targets: MAPTAPPSNCA
    SLEEP-AGE-ELDERLY-4
    Sleep disorder (subtype) – elderly variant 4
    G47.8
    Targets: MAPT
    SLEEP-AGE-ELDERLY-5
    Sleep disorder (subtype) – elderly variant 5
    G47.9
    Targets: MAPTAPP
    SLEEP-AGE-ELDERLY-6
    Sleep disorder (subtype) – elderly variant 6
    G47.0
    Targets: MAPTAPPSNCA

    No diseases match your search.

    Try a different keyword, ICD-10 prefix, or target gene name.

    Pipeline mode A

    Start with the disease.

    Pick one of 791 indications. The Engine harvests the targets, builds the compound library, and runs all 65 gates against that disease's biology — automatically.

    1

    Pick the disease

    Type a name or ICD-10 — Glioblastoma, Alzheimer's, Rett syndrome, anything in the catalog.

    2

    Auto-harvest targets

    Open Targets ≥0.4 association + STRING-PPI hop-2 + UniProt cross-refs build the target list — no manual seed needed.

    3

    Auto-build the library

    ChEMBL bioactivity (pchembl ≥4.0) for the top targets, plus PubChem CNS focus set, plus DrugBank ATC N0x — deduplicated by InChIKey.

    4

    Run all 65 gates

    BBB filter first, then ADMET, then docking, then QM/MM scoring — fail-fast at every step. Final dossier in days, not quarters.

    Pipeline mode B

    Start with the target.

    Already know the protein? Type its symbol or UniProt ID. The Engine harvests every known ligand, generates new ones, and runs all 65 gates around that single target.

    1

    Pick the target

    Symbol (EGFR, SNCA, GBA1, LRRK2, MAPT, BACE1…) or UniProt accession — anything druggable.

    2

    Resolve structure

    UniProt → AlphaFold v6 + every PDB drug-bound template the target has. Class-aware: kinase, GPCR, ion-channel each handled differently.

    3

    Harvest + generate ligands

    Pull every known active from PubChem and ChEMBL, expand chemical space with structure-aware generation, and dedup.

    4

    Run all 65 gates around the target

    Same gate sequence as a disease run — but every result is anchored to your one target. Top-10 lead dossier ranked by ΔG, BBB, safety, and novelty.

    The translation gap

    10 species. One human prediction.

    A drug that works in a mouse fails in humans 89% of the time. We close that gap by simulating across the entire evolutionary tree — and combining the signals into a single human-translation score.

    Human
    Homo sapiens
    The clinical target.
    Mouse
    Mus musculus
    Default preclinical model.
    Rat
    Rattus norvegicus
    PK/Tox gold standard.
    Dog
    Canis familiaris
    Cardiac safety and veterinary CNS.
    Pig
    Sus scrofa
    Closest large-animal BBB anatomy.
    Macaque
    Macaca mulatta
    Final translation gate before IND.
    Marmoset
    Callithrix jacchus
    Small primate · scalable CNS phenotyping.
    Zebrafish
    Danio rerio
    Transparent larval BBB · high-throughput imaging.
    Fly
    Drosophila melanogaster
    Genetics powerhouse.
    C. elegans
    Caenorhabditis elegans
    Neuron-by-neuron mapped.

    How we combine them

    1

    Per-species PK simulation

    PBPK with species-specific brain compartments + transporter expression.

    2

    Ortholog conservation

    Sequence + structural conservation of the target between species.

    3

    Bayesian fusion → TRS

    Each species' signal becomes a posterior. We fuse them into a single Translation Reliability Score with a calibrated confidence interval — that's the number that goes in the dossier.

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