Muscular dystrophy includes more than 30 genetic muscle diseases, with Duchenne and Becker the most common. Gene therapy is finally reaching patients — elevidys, a one-time gene therapy for Duchenne, is approved, and exon-skipping drugs and gene-editing approaches are advancing.
What's actually going on in research
Trials are testing gene therapies for Duchenne and other forms, exon-skipping drugs that target specific genetic mutations, treatments to preserve heart and lung function, and approaches for less common dystrophies like limb-girdle and facioscapulohumeral. Researchers are also studying steroids alternatives and physical therapy strategies.
Gene therapy
Elevidys delivers a working copy of a shortened dystrophin gene and is the first gene therapy for Duchenne. Newer constructs and longer-lasting delivery are in trials.
Exon-skipping drugs
Drugs like eteplirsen, golodirsen, and viltolarsen target specific Duchenne mutations and let cells produce a partial dystrophin protein. New options are advancing for additional mutations.
Heart and lung care
Most boys with Duchenne now live into adulthood, making cardiac and respiratory care critical. Trials are testing treatments to preserve heart function and breathing.
What to know before you search
Eligibility often depends on type of muscular dystrophy, specific genetic mutation, age, current function, and heart and lung status.
What types of trials are currently open
- Gene therapy trials — Testing one-time gene therapies for Duchenne and other muscular dystrophies.
- New medication trials — Testing exon-skipping drugs, steroid alternatives, and other medications for muscular dystrophy.
- Heart and lung trials — Testing treatments to preserve cardiac and respiratory function.
- Therapy strategy trials — Testing combinations of treatments and physical therapy approaches.
- Observational studies — Following people with muscular dystrophy to understand progression and identify markers for trials.
Recently added Muscular Dystrophy trials
Amino Acids and Exercise in FSHD
This study evaluates whether amino acid supplementation, combined with a structured diet and exercise program, improves body composition (increased muscle mass and reduced fat mass) and physical performance in individuals with facioscapulohumeral muscular dystrophy (FSHD). The study also compares responses to the intervention between individuals with FSHD and healthy individuals matched for age and sex.
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility for more details) to control and receive feedback from assistive devices.
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