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Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy
Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy
Avidity Biosciences Reports Positive Data Demonstrating AOC 1044 Delivers Unprecedented Concentrations of PMO in Muscle Following a Single Dose in Healthy Volunteers from Phase 1/2 EXPLORE44™ Trial for Duchenne Muscular Dystrophy
As an early funder of Avidity Biosciences, we are pleased to share that AOC 1044, designed to skip exon 44 in the dystrophin gene, produced significant exon skipping in healthy volunteers.
Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial
Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility Analysis
Navigating Duchenne treatment: AGAMREE & Catalyst Pathways
Join us for this pre-recorded presentation and Q&A discussing to learn about a new FDA-approved treatment for Duchenne muscular dystrophy. During the session, Catalyst Pharmaceuticals will provide an overview of […]
Astellas study results are available for ASP0367 (MA-0211) in Pediatric Male patients with Duchenne Muscular Dystrophy (0367-CL-0102)
Read Astellas community letter below:
Solid Biosciences received clearance by the U.S. FDA for an Investigational New Drug (IND) application for SGT-003 for DMD
Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
CureDuchenne Raises More Than $1.3 Million at Ninth Annual Napa in Newport to Help Find a Cure for Duchenne Muscular Dystrophy
Southern California’s Premier Wine Event Raised Funds and Awareness for Rare Disease with Acclaimed Napa Valley Vintners. NEWPORT BEACH Calif., November 14, 2023 – CureDuchenne and Vintner Chair Melka Estates hosted the Ninth Annual Napa […]
Read and Watch These Four Stories About Disability This Holiday Season
The silver screen has been featuring more and more stories about people with disabilities, whether it be developmental, physical, or mental. With more representation in popular culture, comes more understanding […]
Community Letter: EMBARK Part 1 Topline Results
Sarepta Therapeutics announced topline results from EMBARK