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🗺️ Disease Atlas · Nervous system disorder

Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by rapidly progressive muscle weakness and wasting due to degeneration of skeletal, smooth and cardiac muscle.

🔔 Follow weekly
🧬2530genes / proteins with evidence
💊62drugs and clinical candidates
🧪12+recruiting trials worldwide
✅9approved
📈18phase 3
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Understand Duchenne muscular dystrophy

Genetic diseases · One changed letter in the DNA
🎓 Learn more

What it is

Muscular dystrophy (MD) is a group of more than 30 genetic diseases. They cause weakness of the muscles. Over time, the weakness gets worse and can cause trouble walking and doing daily activities. Some types of MD can also affect other organs.

What are the types of muscular dystrophy (MD)?

There are many different types of MD. Some of the more common types include:

From MedlinePlus: Muscular Dystrophy (US National Library of Medicine) ↗

🧪 Genetic diseases: Genetic diseases are caused by a change in a gene (or a chromosome), often inherited from the parents - such as sickle cell disease, cystic fibrosis and Duchenne muscular dystrophy.

❓ Why it happens

  • A gene is a recipe for a protein. A change in its DNA letters can make the protein faulty or missing.
  • The change can be inherited from one or both parents, or happen for the first time in a child.

🫀 What it does to the body

  • In sickle cell disease, a change in the haemoglobin gene bends red blood cells into a sickle shape; they get stuck in small blood vessels, causing pain, anaemia and organ damage.

🧱 Why it is hard to cure

  • Fixing a gene safely in the right cells of the body is technically difficult.
  • Gene therapies are very expensive, and many genetic diseases are rare, with few patients for trials.

🚀 What research is trying now

  • Gene therapies that add a working copy of a gene.
  • CRISPR gene editing, now approved for sickle cell disease and beta thalassaemia.
  • RNA medicines that work around a faulty gene.

General information about genetic diseases, reviewed by people; it does not describe any one person. Below: the evidence for Duchenne muscular dystrophy itself.

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AI briefing

Where the science on Duchenne muscular dystrophy 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 Duchenne muscular dystrophy.

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Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
DMD dystrophinPROTACantibody🧊 AlphaFold structure ↗ 0.87
NR3C1 nuclear receptor subfamily 3 group C member 1PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.59
NR3C2 nuclear receptor subfamily 3 group C member 2PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.56
HDAC4 histone deacetylase 4PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.53
HDAC9 histone deacetylase 9PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.53
HDAC2 histone deacetylase 2PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC11 histone deacetylase 11PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC8 histone deacetylase 8PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC6 histone deacetylase 6PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC5 histone deacetylase 5PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC3 histone deacetylase 3PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC7 histone deacetylase 7PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC1 histone deacetylase 1PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
HDAC10 histone deacetylase 10PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.52
RPS6 ribosomal protein S6PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.51

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

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Drugs and candidates

Every medicine linked to Duchenne muscular dystrophy, by how far it has got
Approved · 9
Ataluren80S Ribosome modulatorApproved
CasimersenDystrophin pre-mRNA positive modulatorApproved
DeflazacortGlucocorticoid receptor agonistApproved
EteplirsenDystrophin pre-mRNA positive modulatorApproved
GivinostatHistone deacetylase inhibitorApproved
Givinostat HydrochlorideHistone deacetylase inhibitorApproved
Show all 9
GolodirsenDystrophin pre-mRNA positive modulatorApproved
VamoroloneGlucocorticoid receptor agonist; Mineralocorticoid receptor antagonistApproved
ViltolarsenDystrophin pre-mRNA positive modulatorApproved
In phase 2 / 3 · 20
CreatinePhase 3
Delpacibart ZotadirsenPhase 3
DeramiocelPhase 3
DrisapersenDystrophin pre-mRNA positive modulatorPhase 3
EdasalonexentNuclear factor NF-kappa-B complex inhibitorPhase 3
EnalaprilAngiotensin-converting enzyme inhibitorPhase 3
Show all 20
EplerenoneMineralocorticoid receptor antagonistPhase 3
Fordadistrogene MovaparvovecPhase 3
GlutaminePhase 3
IdebenonePhase 3
L-CitrullinePhase 3
MetforminMitochondrial complex I (NADH dehydrogenase) inhibitor; Mitochondrial glycerol-3-phosphat…Phase 3
PamrevlumabConnective tissue growth factor inhibitorPhase 3
PrednisoneGlucocorticoid receptor agonistPhase 3
SpironolactoneMineralocorticoid receptor antagonistPhase 3
TadalafilPhosphodiesterase 5A inhibitorPhase 3
TamoxifenEstrogen receptor alpha modulatorPhase 3
UbidecarenonePhase 3
Epigalocatechin GallatePhase 2/3
SuvodirsenDystrophin pre-mRNA positive modulatorPhase 2/3
Early trials · 17
(+)-EpicatechinPhase 1/2
(-)-EpicatechinPhase 1/2
CanakinumabInterleukin-1 beta inhibitorPhase 1/2
MecaserminInsulin-like growth factor I receptor agonistPhase 1/2
PentoxifyllineAdenosine A2 receptor antagonist; 3',5'-cyclic phosphodiesterase inhibitorPhase 1/2
Sodium ChloridePhase 1/2
Show all 17
TacrolimusFK506-binding protein 1A inhibitorPhase 1/2
ArgininePhase 1
BocidelparPhase 1
Delandistrogene MoxeparvovecDystrophin exogenous genePhase 1
FilgrastimGranulocyte colony stimulating factor receptor agonistPhase 1
FlavocoxidPhase 1
GentamicinBacterial 70S ribosome inhibitorPhase 1
ImlifidasePhase 1
PrednisoloneGlucocorticoid receptor agonistPhase 1
RimeporidePhase 1
SildenafilPhosphodiesterase 5A inhibitorPhase 1
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AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy