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Home › Conditions › Muscular Dystrophies

Muscular Dystrophies

Subtypes of Muscular Dystrophies

Duchenne muscular dystrophy 4 Facioscapulohumeral Muscular Dystrophy 1

5 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Muscular Dystrophies

9 trials tracked for Muscular Dystrophies: 3 in phase 3 or 4 and 3 with published results. The most-cited published study has 455 citations.

9Trials tracked
3Phase 3 & 4
0Recruiting
3With published results
Phase distribution
Phase 3 3 Phase 2 2 Phase 1 2 Other / NA 2
  1. Phase 3 Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Completed · 455 cited
  2. Phase 3 A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy Completed · 127 cited
  3. Phase 3 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy Completed
  4. Phase 2 A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) Completed
  5. Phase 2 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy Completed
  6. Phase 1 First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect Completed
Show 3 more trials
  1. Phase 1 Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy Completed
  2. N/A Transmembrane Electromyography (TM-EMG) for the Assessment of Neuromuscular Function in the Oropharynx Completed
  3. N/A The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities Completed

Showing the 9 most-cited and recently-updated of 9 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Muscular Dystrophies: what the trials found

Ataluren demonstrated a statistically significant improvement in the 6-Minute Walk Distance (6MWD) at week 72 when analyzed using an Intent-to-Treat (ITT) population (-53.01; -67.43, p=0.0248) 1. However, this same metric did not reach statistical significance in the modified Intention-to-treat (mITT) population 1. In a separate trial of Ataluren, no significant change from baseline was observed in 6MWD at week 48 (p=0.213) 2.

GSK2402968 (6mg/kg/week) did not show statistically significant improvements in muscle function as measured by the 6-Minute Walk Distance (p=0.415), the Linearized North Star Ambulatory Assessment total score (p=0.757), or the 4 Stair Climb Ascent Velocity (p=0.718) at week 48 3.

Recent results — preliminary, needs further review

  • CAP-1002 showed a change from baseline in functional capacity as assessed by the PUL Version 1.2 mid-level dimension score at month 12 (65.5; 29.3) 4.
  • GSK2402968 (3mg/kg/week) did not show statistically significant changes in 6MWD, Rise From Floor time, or 4 Stair Climb Ascent/Descent time at week 24 5.
  • GSK3439171A demonstrated steady state accumulation ratios of 1.07 (5 mg), 1.21 (11 mg), and 1.16 (40 mg) 6.

For the clinician treating this condition

  • Ataluren showed statistically significant improvement in 6MWD at week 72 specifically within the ITT population, though results were not significant in the mITT cohort 1.
  • GSK2402968 (6mg/kg/week) did not produce significant improvements across multiple functional mobility metrics including 6MWD and North Star Ambulatory Assessment scores 3.

AI synthesis of 6 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Transition programs for DMD vary widely with significant gaps in policy-driven implementation and neurodiversity support
Primary Care & Family Medicine Meta-analysis
Transition programs for DMD vary widely with significant gaps in policy-driven implementation and neurodiversity support Health systems for young people with Duchenne muscular dystrophy need better support and clear rules
This systematic review with narrative synthesis examines transition-related health systems for youth and adults living with Duchenne muscula…
A review of 42 studies shows care plans for teens with Duchenne muscular dystrophy vary widely. Most programs are temporary projects instead…
Frontiers May 29, 2026
Russian FSHD registry shows moderate inverse correlation between D4Z4 repeats and clinical severity
Genetics & Precision Medicine Cohort
Russian FSHD registry shows moderate inverse correlation between D4Z4 repeats and clinical severity What does FSHD look like in Russia? A registry study maps the disease
An observational cohort study of 470 patients in the Russian FSHD Patient Registry found genetic confirmation in 76% of participants.
Nearly half of Russian FSHD patients first notice shoulder blade weakness, while others start with facial symptoms or a classic childhood fo…
medRxiv Apr 8, 2026
Tamoxifen adjunct to corticosteroids shows no motor benefit in Duchenne muscular dystrophy extension study
Pediatrics RCT
Tamoxifen adjunct to corticosteroids shows no motor benefit in Duchenne muscular dystrophy extension study Does adding tamoxifen to standard treatment help boys with Duchenne muscular dystrophy?
A 48-week open-label extension of a phase 3 trial in 66 boys with DMD found no significant difference in motor function change between early…
Adding tamoxifen to standard steroid treatment did not improve motor function or slow disease progression in boys with Duchenne muscular dys…
Apr 3, 2026
Phase 3 RCT: Ataluren shows favorable trend for slowing functional decline in nonsense mutation DMD
Pediatrics RCT
Phase 3 RCT: Ataluren shows favorable trend for slowing functional decline in nonsense mutation DMD Can a drug help boys with Duchenne muscular dystrophy walk longer?
A phase 3 RCT of 359 people with nonsense mutation Duchenne muscular dystrophy found that 72 weeks of ataluren treatment was associated with…
Boys with a specific genetic form of Duchenne muscular dystrophy who took ataluren saw their walking ability decline less over 72 weeks than…
Apr 3, 2026
Phase 2 trial of oral ifetroban in DMD cardiomyopathy reports no safety results yet
Cardiology Phase II
Phase 2 trial of oral ifetroban in DMD cardiomyopathy reports no safety results yet Can a new pill help protect the hearts of boys with Duchenne muscular dystrophy?
A phase 2 randomized, double-blind, placebo-controlled trial evaluated low- and high-dose oral ifetroban in 46 males with DMD-associated car…
A new pill called ifetroban is being tested to safely treat heart weakness in boys with Duchenne muscular dystrophy, addressing a leading ca…
CT.gov Apr 2, 2026