Neuromuscular

Duchenne Muscular Dystrophy

Also known as DMD, Duchenne MD, dystrophin deficiency

Duchenne muscular dystrophy is caused by mutations in the DMD gene that prevent production of functional dystrophin, a protein essential for muscle membrane stability. Progressive muscle degeneration leads to loss of ambulation typically by

ORPHA:98896 ↗Gene DMD (dystrophin)Prevalence 1-9 per 100,000 (Orphanet)Onset ChildhoodGenetic (X-linked recessive)

61

studies recruiting now

as of 7 Sept 2026

497

studies registered in total

as of 7 Sept 2026

11

countries with a recruiting site

as of 7 Sept 2026

17 Aug 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 61 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Patient organisations

Parent Project Muscular DystrophyPatient association
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Registry: TREAT-NMD DMD Global Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is caused by mutations in the DMD gene that prevent production of functional dystrophin, a protein essential for muscle membrane stability. Progressive muscle degeneration leads to loss of ambulation typically by age 12, followed by cardiac and respiratory involvement. Multiple exon-skipping therapies and one gene therapy have received FDA approval. Becker MD is the milder allelic form where some dystrophin is produced.

Common clinical features

Progressive muscle weaknessElevated circulating creatine kinaseSkeletal muscle atrophyCalf muscle hypertrophyWaddling gaitProximal muscle weaknessScoliosisCardiomyopathy

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Before you apply

Things trial teams commonly ask about for Duchenne Muscular Dystrophy. Not eligibility rules; those are set by each study.

  • Specific DMD mutation (exon deletion, duplication, or point mutation) is essential - many trials target specific exons (e.g., exon 51, 53)
  • Ambulatory status (walking vs. non-ambulatory) divides most trial populations
  • Corticosteroid use (deflazacort, prednisone, or vamorolone) and history is typically required at baseline

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).