The Muscular Dystrophy Association is hosting their 2025 Clinical & Scientific Conference meeting March 16 – 19 in Dallas, Texas. Several companies doing Duchenne muscular dystrophy will be presenting data from their current clinical trials. Defeat Duchenne Canada is pleased to share important updates as they come through for our Canadian Duchenne audience.
Avidity Biosciences
Avidity Biosciences is pleased to present new positive del-zota topline data are being presented this week at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference consistent with the positive initial del-zota data they shared in August 2024, including statistically significant improvements in dystrophin production, exon skipping and creatine kinase levels as well as favorable safety and tolerability across the dose cohorts. Please see their community letter below with additional details about the study.
Dyne Therapeutics
Dyne Therapeutics shared new long-term clinical data from the ongoing Phase 1/2 DELIVER trial of DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. These most recent data demonstrated sustained functional improvement at the selected registrational dose of 20 mg/kg every four weeks (approximate PMO dose) and a continued favorable safety profile. The full press release may be viewed at the link below.
REGENXBIO
REGENXBIO shared updates on RGX-202, their investigational gene therapy for DMD, reflecting exciting progress in our AFFINITY DUCHENNE® trial.
- The Phase 3 portion of this trial is open and enrolling patients.
- The AFFINITY DUCHENNE trial of RGX-202 is nearly 50% enrolled.
- REGENXBIO expects to share:
- REGENXBIO expects to submit a BLA in mid-2026 in the U.S. under the accelerated approval pathway.
- Additional Phase 1/2 data, including new functional data, in the first half of 2025.
- Top line pivotal data in the first half of 2026.
- New biomarker data from Phase 1/2 at the MDA conference next week, including the first data from the cohort of patients aged 1-3.
Please check back throughout the week to learn about other highlights from the 2025 MDA Clinical & Scientific Conference.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: