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Disease

Muscular dystrophy

Late-stage therapeutic development
18
Clinical trials
12
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q2 2028.
  • 8 industry developments reported.
Upcoming Milestones
Clinical Milestones7View
Results posted 1
Results expected 4
Recently completed 2
Industry & Market8View all 8
+3 more in the activity timeline below
Activity timeline15

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Atalurenapproved

Product authorisation ended — Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a… (2025)

Vamoroloneapproved

Original application approval — Agamree is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients a… (2023)

Clinical trials

13 sponsors · 2 new · 2 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme
18
All trials
6
Active
18
Late-stage
6
Completed
Late-stage studies

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy

Phase 3 · Recruiting · Solid Biosciences Inc.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping

Phase 3 · Recruiting · Dyne Therapeutics

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants With Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)

Phase 3 · Active, not recruiting · ML Bio Solutions, Inc.

Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

Phase 4 · Active, not recruiting · NS Pharma, Inc.

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Phase 3 · Recruiting · Avidity Biosciences, Inc.

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

Phase 3 · Active, not recruiting · Sarepta Therapeutics, Inc.
Recruiting

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy

Phase 3 · Recruiting · Solid Biosciences Inc.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping

Phase 3 · Recruiting · Dyne Therapeutics

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Phase 3 · Recruiting · Avidity Biosciences, Inc.
Recently completed

A Phase 3, Randomized, Placebo-controlled, Double-blind and Open-label, Extension Study of TAS-205 in Patients With Duchenne Muscular Dystrophy

Phase 3 · Completed · Taiho Pharmaceutical Co., Ltd.

An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Phase 3 · Completed · PTC Therapeutics

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Phase 3 · Completed · Sarepta Therapeutics, Inc.

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

Phase 3 · Completed · NS Pharma, Inc.

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Phase 3 · Completed · PTC Therapeutics

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy

Phase 3 · Completed · Catabasis Pharmaceuticals

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

Product approvals and authorisations
2023emaOriginal application approval — Vamorolone· Agamree is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older.  source ↗
Other regulatory activity
2025emaProduct authorisation ended — Ataluren· Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

DMD0.66CAPN30.50POMT20.47SGCA0.43SELENON0.43ANO50.42JAG20.37DTNA0.37PYROXD10.37HNRNPA2B10.37UBE2E30.30EXOC6B0.30

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Dystrophinvia DMD0.66
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.