Duchenne muscular dystrophy (DMD) is a severe X-linked genetic disease caused by mutations in the dystrophin gene, leading to progressive muscle breakdown that typically confines boys to wheelchairs by their early teens and affects the heart and breathing muscles over time. It is the most common and serious form of muscular dystrophy in children.
What's actually going on in research
Several exon-skipping drugs have been granted accelerated or conditional approval to restore partial dystrophin production in specific mutation subsets, and a gene therapy — delandistrogene moxeparvovec — has received approval in the US for younger children. Trials are testing more potent exon-skipping drugs, CRISPR-based gene correction, utrophin upregulators, and anti-inflammatory or muscle-preserving agents to complement or extend the reach of these advances.
Gene replacement therapy
Delandistrogene moxeparvovec delivers a shortened but functional micro-dystrophin gene via an AAV vector and is approved for ambulatory children under five; trials are extending this to older and non-ambulatory patients.
Exon-skipping drugs
Antisense oligonucleotides that skip specific exons to restore the dystrophin reading frame are approved for some mutations, and next-generation peptide-conjugated versions with improved muscle delivery are in active trials.
CRISPR gene correction
Early-phase trials are testing CRISPR-based approaches to permanently correct dystrophin mutations in muscle and cardiac cells, aiming for a one-time treatment that restores more complete protein function.
What to know before you search
Eligibility depends on the specific dystrophin mutation, age, ambulatory status, and prior corticosteroid or gene therapy use.
What types of trials are currently open
- Gene therapy trials — Testing AAV-delivered micro-dystrophin constructs across different age groups and mutation types.
- Exon-skipping trials — Evaluating antisense drugs and next-generation conjugates that restore dystrophin reading frame for specific mutations.
- CRISPR trials — Early-phase studies of gene-editing approaches to permanently correct the dystrophin gene.
- Cardioprotection trials — Studying drugs to slow or prevent the cardiomyopathy that develops in most DMD patients.
- Anti-inflammatory trials — Testing corticosteroid alternatives and other anti-inflammatory drugs to slow muscle degeneration.
Recently added Duchenne 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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