Becker muscular dystrophy (BMD) is a milder X-linked dystrophin deficiency than Duchenne, caused by in-frame mutations that allow production of a shortened but partially functional dystrophin protein. Onset and progression vary widely — some men remain ambulatory into their 40s while others lose mobility in their 20s — and heart involvement is a leading cause of complications.
What's actually going on in research
Unlike Duchenne, BMD has no approved disease-modifying therapy, making it a target for treatments developed for DMD that may also benefit Becker patients. Trials are testing exon-skipping drugs, micro-dystrophin gene therapy, and utrophin upregulators. Cardiac monitoring and heart failure treatment are a focus given that cardiomyopathy often progresses independent of skeletal muscle severity in BMD.
Gene therapy extension
Micro-dystrophin gene replacement therapies being developed for DMD are being evaluated in BMD patients with advanced disease, where boosting dystrophin levels may slow further decline.
Utrophin upregulation
Small molecules that increase production of utrophin — a functional substitute for dystrophin — are in trials and could benefit all dystrophin-deficiency patients regardless of specific mutation.
Cardiac management
Trials are studying angiotensin-converting enzyme inhibitors, beta-blockers, and other agents to slow cardiomyopathy progression in BMD, where heart disease often drives morbidity independently of muscle status.
What to know before you search
Eligibility depends on the specific dystrophin mutation, cardiac function, ambulatory status, and whether the patient has already been enrolled in DMD gene therapy trials.
What types of trials are currently open
- Gene therapy trials — Testing AAV micro-dystrophin delivery in Becker patients with functional decline.
- Utrophin upregulator trials — Evaluating small molecules that increase the natural dystrophin substitute protein across mutation types.
- Cardiac trials — Studying drugs to prevent or slow cardiomyopathy in Becker muscular dystrophy.
- Exon-skipping trials — Testing antisense approaches in Becker patients amenable to exon skipping based on their specific mutations.
- Natural history trials — Characterizing disease progression and biomarkers to support future drug development.
Recently added Becker Muscular Dystrophy trials
Duchenne Electronic Health Record Study
This study aims to collect retrospective and prospective, long-term data of patients with dystrophinopathy (including Duchenne, Becker, and female carriers) through electronic transfer. At select clinics across the United States, electronic health record (EHR) data from consented patients will be pushed into PPMD's Duchenne Outcomes Research Interchange (the Interchange), where the EHR data can be combined with patient-reported data from The Duchenne Registry. By combining this data in a central hub, we will gain a more complete picture of Duchenne and Becker muscular dystrophy, allowing researchers and clinicians to develop treatments faster and to improve and refine the standards of care for Duchenne and Becker. The ultimate goal is to optimize function, quality of life, and survival of Duchenne and Becker patients. EHR data collected will be fully identifiable retrospective data for core clinical data elements going back ten years (as available) from the date of consent; going back one year for retrospective clinical notes from the date of consent; and prospectively collecting both core clinical data elements and clinical notes. Information collected will align with the FHIR U.S. core data elements, also known as the Common Clinical Data Set. PPMD partnered with Prometheus Research (an IQVIA company), an industry leader in health data informatics, to launch both the EHR Study and the Interchange. All data is stored securely and in accordance with strict industry standards and patient privacy laws. Participation in the EHR data extraction is voluntary, and a patient can withdraw consent at any time.
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
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