Condition

Muscular Dystrophies clinical trials and research

• 11Clinical trials
• 11NIH research projects
• 11Countries

Newest Muscular Dystrophies clinical trials

All recruiting trials (11)

• Zeleciment Rostudirsen (DYNE-251) for Duchenne Muscular Dystrophy in Ambulatory Boys

  This trial tests whether intravenous Zeleciment Rostudirsen (DYNE-251) given every 4 weeks is safe and helps improve function in ambulatory boys with Duchenne muscular dystrophy who have a specific dystrophin gene mutation.

  Recruiting Phase 3 Dyne Therapeutics · Hillsborough, North Carolina · NCT07608432

• AOC 1044 in Duchenne Muscular Dystrophy with Exon 44 Skipping

  This study tests whether intravenous AOC 1044 helps boys with Duchenne muscular dystrophy caused by mutations amenable to exon 44 skipping.

  Recruiting at 20 sites Phase 3 Avidity Biosciences, Inc. · Westmead, New South Wales and 19 other locations · NCT07587242

• Virtual Park for Children: Dual-Task Training with Virtual Reality

  This study tests whether a virtual reality, dual-task cycling program can slow motor decline and boost engagement for kids with neuromuscular diseases compared with standard therapy.

  Recruiting at 3 sites Istituto di Sistemi e Tecnologie Industriali Intelligenti per il Manifatturiero Avanzato · Bosisio Parini, LC and 3 other locations · NCT07580365

• Implanted brain interface to let people with severe paralysis control assistive devices

  It will try an implanted brain interface that records and stimulates brain signals so adults with severe paralysis can control assistive devices and receive sensory feedback.

  Recruiting Johns Hopkins University · Baltimore, Maryland · NCT07521930

• Measuring muscle health with a handheld electrical impedance device (mScan)

  This project will test whether a handheld mScan device can quickly measure muscle health in adults with myopathies and in people without muscle disease by comparing its readings to MRI.

  Recruiting Beth Israel Deaconess Medical Center · Boston, Massachusetts · NCT07502989

• Reducing fear of falling in people with muscular dystrophy

  This study tests whether adding cognitive-behavioral therapy to motor rehabilitation helps people aged 16–65 with muscular dystrophy who have a persistent fear of falling.

  Recruiting Universita di Verona · Verona, verona · NCT07129954

• Natural history study of LAMA2 muscular dystrophy in children

  This study is trying to understand how LAMA2 muscular dystrophy affects children over time to help improve future treatments.

  Recruiting Hospital Universitari Vall d'Hebron Research Institute · Barcelona, Barcelona · NCT06924125

• Observing walking patterns in patients with neuromuscular diseases

  This study is testing how walking patterns change in people with neuromuscular diseases and whether wearable sensors can help track their daily activity and fatigue levels.

  Recruiting IRCCS Eugenio Medea · Bosisio Parini, Lecco and 1 other locations · NCT06666816

• Using a brain-computer interface to help people with speech difficulties communicate

  This study is testing a brain-computer interface to help people who can't speak due to conditions like ALS communicate by turning their thoughts into speech.

  Recruiting Massachusetts General Hospital · Sacramento, California · NCT06094205

• Using a neural interface to help people with tetraplegia control devices with their thoughts

  This study is testing a new brain interface that helps people with tetraplegia, especially those with ALS, control devices like computers just by thinking about it.

  Recruiting at 2 sites Massachusetts General Hospital · Stanford, California and 1 other locations · NCT05724173

• Using brain signals to control devices for movement and speech

  This study is testing if people with severe neurological disorders can use brain signals to control devices that help them move and speak better.

  Recruiting University of California, San Francisco · San Francisco, California · NCT03698149

NIH-funded research (11)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Noninvasive breathing imaging to guide care for people with muscular dystrophy

  A noninvasive imaging tool called electrical impedance tomography (EIT) will be studied to help decide when to start noninvasive ventilation for people with muscular dystrophy.

  NIH research Colorado State University · Fort Collins, United States · NIH-11415735

• Bicistronic gene therapy for limb girdle muscular dystrophy type R9

  A patient-facing overview of a first-in-class gene therapy approach using two genes delivered by AAV to help people with LGMDR9.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11397452

• Understanding why scarring happens in muscular dystrophy muscles

  Researchers are studying why scar tissue forms in muscles affected by muscular dystrophy and how to slow or reverse it in patients.

  NIH research University of Florida · Gainesville, United States · NIH-11370556

• Addressing Shortcomings of AAV Gene Therapies for Muscular Dystrophy

  A national research center is working to improve AAV-based gene therapies for Duchenne and related muscular dystrophies so that patients may get safer, more effective treatments.

  NIH research University of Florida · Gainesville, United States · NIH-11310128

• Two-gene AAV therapy for Limb-Girdle Muscular Dystrophy R9

  Developing a two-gene AAV gene therapy to replace the faulty gene and rebuild muscle for people with LGMDR9 (Limb Girdle Muscular Dystrophy Type R9).

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11169982

• Improving Gene Therapies for Muscular Dystrophies

  This center is working to improve gene therapies for Duchenne and Limb Girdle Muscular Dystrophy, aiming to make them safer and more effective for patients.

  NIH research University of Florida · Gainesville, United States · NIH-11130228

• Stopping Muscle Scarring in Muscular Dystrophies

  This project explores how muscle scarring, called fibrosis, develops in muscular dystrophies like Duchenne MD, hoping to find new ways to help muscles heal better.

  NIH research University of Florida · Gainesville, United States · NIH-11092236

• Understanding Muscle Weakness in Muscular Dystrophies

  This research explores how certain proteins called actins help maintain muscle structure and function in people with muscular dystrophy.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11047663

• Understanding how caspases affect muscle development and disorders

  This study is looking at how certain enzymes, called caspases, affect muscle growth and breakdown, especially in people with muscular dystrophies, to better understand how they might help or hurt muscle development.

  NIH research Univ of North Carolina Chapel Hill · Chapel Hill, United States · NIH-10992642

• Gene therapy for Limb Girdle Muscular Dystrophy Type R9

  This study is testing a new gene therapy for people with Limb Girdle Muscular Dystrophy Type R9 that aims to replace the faulty gene and help rebuild muscle strength, with hopes of improving muscle function and slowing down the disease.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-10992526

• Creating improved gene therapy vectors for muscular dystrophies

  This study is working on improving a type of virus used in gene therapy to help treat Duchenne muscular dystrophy (DMD) by making it safer and more effective, so that patients can benefit from better treatments with fewer side effects.

  NIH research University of Florida · Gainesville, United States · NIH-10768732

Related topics

Quick answers

How many Muscular Dystrophies clinical trials are recruiting right now?

11, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 11 countries. The most sites are in United States (6 trials), Italy (4 trials), Spain (2 trials), Australia (1 trial), Belgium (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 2. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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