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🗺️ Disease Atlas · Nervous system disorder

Parkinson disease

A progressive degenerative disorder of the central nervous system characterized by loss of dopamine producing neurons in the substantia nigra and the presence of Lewy bodies in the substantia nigra and locus coeruleus. Signs and symptoms include tremor which is most pronounced during rest, muscle rigidity, slowing of the voluntary movements, a tendency to fall back, and a mask-like facial expression.

🔔 Follow weekly
🧬8994genes / proteins with evidence
💊333drugs and clinical candidates
🧪12+recruiting trials worldwide
✅57approved
📈44phase 3
🧠

Understand Parkinson disease

Brain and nerves · Nerve cells that slowly stop working
🎓 Learn more

What it is

Symptoms begin gradually, often on one side of the body. Later they affect both sides. They include:

Trembling of hands, arms, legs, jaw and face

Stiffness of the arms, legs and trunk

From MedlinePlus: Parkinson's Disease (US National Library of Medicine) ↗

🧠 Brain and nerves: In neurodegenerative diseases, nerve cells in the brain or spinal cord stop working and die over time - affecting memory (Alzheimer's disease), movement (Parkinson's disease) or muscles (ALS).

❓ Why it happens

  • Proteins fold the wrong way and clump together inside or between nerve cells - amyloid and tau in Alzheimer's disease, alpha-synuclein in Parkinson's disease.
  • Inflammation, some genes (such as APOE4) and, above all, age raise the risk.

🫀 What it does to the body

  • As connections between nerve cells are lost, memory, thinking, movement or speech fade.
  • The changes start years - often decades - before the first symptoms.

🧱 Why it is hard to cure

  • Most medicines cannot easily pass the blood-brain barrier that protects the brain.
  • By the time symptoms appear, many nerve cells are already lost, and they do not grow back.
  • Many drug trials have failed, partly because the diseases are found late.

🚀 What research is trying now

  • Antibodies that clear amyloid and modestly slow early Alzheimer's disease (approved in some countries).
  • Blood tests that detect Alzheimer's changes years earlier.
  • Gene-targeted medicines, such as one approved in the US for an inherited form of ALS.

General information about brain and nerves, reviewed by people; it does not describe any one person. Below: the evidence for Parkinson disease itself.

🤖

AI briefing

Where the science on Parkinson disease stands, from the evidence on this page

AI reads the targets, drugs, trials and papers below and writes where the science stands — every claim cited. It is shared with everyone who opens Parkinson disease.

🎯

Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
LRRK2 leucine rich repeat kinase 2PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.88
ATP13A2 ATPase cation transporting 13A2PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.87
SNCA synuclein alphaPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.87
PRKN parkin RBR E3 ubiquitin protein ligasePROTACsmall molecule🧊 AlphaFold structure ↗ 0.85
PINK1 PTEN induced kinase 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.85
PLA2G6 phospholipase A2 group VIPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.83
DNAJC6 DnaJ heat shock protein family (Hsp40) member C6PROTACantibody🧊 AlphaFold structure ↗ 0.82
PARK7 Parkinsonism associated deglycasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.81
FBXO7 F-box protein 7PROTAC🧊 AlphaFold structure ↗ 0.80
VPS35 VPS35 retromer complex componentPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.79
GBA1 glucosylceramidase beta 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.77
VPS13C vacuolar protein sorting 13 homolog CPROTAC🧊 AlphaFold structure ↗ 0.73
GRIN1 glutamate ionotropic receptor NMDA type subunit 1PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.70
MAPT microtubule associated protein tauPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.68
CHRM1 cholinergic receptor muscarinic 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.67

Green labels: what the target is known to be druggable by. The score combines genetics, somatic mutations, drugs, expression and literature.

💊

Drugs and candidates

Every medicine linked to Parkinson disease, by how far it has got
Approved · 20
AmantadineGlutamate [NMDA] receptor antagonist; Matrix protein 2 inhibitorApproved
Amantadine HydrochlorideGlutamate [NMDA] receptor antagonist; Matrix protein 2 inhibitorApproved
ApomorphineD2-like dopamine receptor agonistApproved
Apomorphine HydrochlorideD2-like dopamine receptor agonistApproved
Atropine SulfateMuscarinic acetylcholine receptor M3 antagonist; Muscarinic acetylcholine receptor M1 ant…Approved
BenztropineMuscarinic acetylcholine receptor M1 antagonistApproved
Show all 20
Benztropine MesylateMuscarinic acetylcholine receptor M1 antagonistApproved
BiperidenMuscarinic acetylcholine receptor M1 antagonistApproved
BornaprineApproved
BromocriptineD2-like dopamine receptor agonistApproved
Bromocriptine MesylateD2-like dopamine receptor agonistApproved
BudipineApproved
CabergolineDopamine D2 receptor agonistApproved
CarbidopaDOPA decarboxylase inhibitorApproved
CycrimineMuscarinic acetylcholine receptor M1 antagonistApproved
DexetimideApproved
Dihydroergocryptine MesylateApproved
DopamineBeta-1 adrenergic receptor agonistApproved
EntacaponeCatechol O-methyltransferase inhibitorApproved
EtanautineApproved
In phase 2 / 3 · 20
AmbroxolPhase 3
AmoxicillinBacterial penicillin-binding protein inhibitorPhase 3
AmpreloxetineNorepinephrine transporter inhibitorPhase 3
AtomoxetineNorepinephrine transporter inhibitorPhase 3
BemdaneprocelPhase 3
BenserazideDOPA decarboxylase inhibitorPhase 3
Show all 20
BuntanetapPhase 3
Buntanetap TartratePhase 3
BuspironeSerotonin 1a (5-HT1a) receptor partial agonistPhase 3
CaffeineAdenosine receptor antagonistPhase 3
ClarithromycinBacterial 70S ribosome inhibitorPhase 3
CreatinePhase 3
DroxidopaAdrenergic receptor agonistPhase 3
DuloxetineSerotonin transporter inhibitor; Norepinephrine transporter inhibitorPhase 3
EszopicloneGABA-A receptor; anion channel positive allosteric modulatorPhase 3
EtilevodopaDopamine D3 receptor agonistPhase 3
ExenatideGlucagon-like peptide 1 receptor agonistPhase 3
FoscarbidopaDOPA decarboxylase inhibitorPhase 3
InosinePhase 3
Lactobacillus AcidophilusPhase 3
Early trials · 20
BecaplerminPlatelet-derived growth factor receptor agonistPhase 1/2
CiforadenantAdenosine A2a receptor antagonistPhase 1/2
DalfampridineVoltage-gated potassium channel blockerPhase 1/2
Darbepoetin AlfaErythropoietin receptor agonistPhase 1/2
Epoetin AlfaErythropoietin receptor agonistPhase 1/2
Epoetin BetaErythropoietin receptor agonistPhase 1/2
Show all 20
FlumazenilGABA-A receptor; anion channel allosteric antagonistPhase 1/2
LiaterminGDNF family receptor alpha-1 agonistPhase 1/2
MethylprednisoloneGlucocorticoid receptor agonistPhase 1/2
MiridesapSerum amyloid P-component cross-linking agentPhase 1/2
SertralineSerotonin transporter inhibitorPhase 1/2
SirolimusFK506-binding protein 1A inhibitorPhase 1/2
TributyrinPhase 1/2
AmlenetugPhase 1
ArecolinePhase 1
Atsm Copper (Ii)Phase 1
BesonprodilPhase 1
BlarcamesinePhase 1
Blarcamesine HydrochloridePhase 1
Bms-984923Phase 1
🛡️

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