Becker muscular dystrophy is a genetic condition caused by mutations in the dystrophin gene, leading to progressive muscle weakness that typically begins in the legs and hips. It's milder and progresses more slowly than Duchenne muscular dystrophy, with symptoms often appearing in the teens or twenties. Treatment focuses on managing heart and breathing complications, preserving mobility, and addressing the underlying dystrophin deficiency.
What's actually going on in research
Trials are testing gene therapies that deliver working copies of micro-dystrophin, exon-skipping drugs to restore some dystrophin production, and gene editing approaches using CRISPR. Researchers are also studying drugs that increase muscle strength, protect the heart, and reduce inflammation. Some therapies developed for Duchenne muscular dystrophy are being adapted for Becker, which shares the same genetic defect but produces a partially functional protein.
Gene therapy
Several trials are delivering micro-dystrophin genes using viral vectors. These shortened but functional versions of dystrophin aim to slow or stop muscle deterioration.
Exon skipping
Drugs that skip over faulty sections of the dystrophin gene can restore some protein production in people with specific mutations. This approach is FDA-approved for Duchenne and being tested in Becker.
Heart protection
Because heart muscle weakening is a major concern in Becker, trials are testing drugs like eplerenone and newer agents to preserve cardiac function. Early treatment may delay or prevent heart failure.
What to know before you search
Eligibility typically depends on confirmed genetic diagnosis, age, baseline muscle and heart function, and whether prior gene therapy has been received.
What types of trials are currently open
- Gene therapy trials — Testing viral vectors that deliver working dystrophin genes to muscle cells, aiming to restore protein production throughout the body.
- Exon-skipping trials — Testing drugs that allow cells to skip over mutated sections of the dystrophin gene and produce a shorter but functional protein.
- Muscle-preserving trials — Testing drugs that reduce inflammation, increase muscle strength, or slow muscle breakdown, often repurposed from other conditions.
- Cardiac trials — Testing treatments to protect heart muscle and delay heart failure, which is a leading cause of complications in Becker muscular dystrophy.
- Natural history studies — Following people with Becker over time to understand how the disease progresses and what factors predict outcomes.
Recently added Becker Muscular Dystrophy trials
Share heart imaging scans to help predict outcomes in Duchenne muscular dystrophy
Dystrophin associated heart dysfunction is a leading cause of death in patients with Duchenne and Becker Muscular dystrophy (DMD/BMD) and Duchenne and Becker muscular dystrophy carriers (MDC); however, the evolution of heart dysfunction is not well-understood. The central objectives of this proposal are to elucidate this evolution of heart dysfunction and identify measures from cardiac MRI images that can predict death or significant heart disease in patients with DMD/BMD/MDC. This study will create a large clinical and cardiac MRI registry of dystrophin associated heart dysfunction, will utilize advanced image analysis techniques, including deep learning neural networks, to comprehensively evaluate every patient, and will create a risk toolkit accessible to clinicians around the world; this proposal has the potential to improve the quality of life in patients with dystrophin associated heart dysfunction by allowing for earlier and more intensive therapy in patients with severe disease and by identifying surrogate outcome measures for use in therapeutic trials.
Receive a gene therapy treatment being tested for Duchenne muscular dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participants will be included in two sequential dose cohorts with increasing doses of the investigational product. Based on the results of Phase I, the dose of the investigational product for use in Phase II will be determined. Phase II is a randomized, single-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
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