Dyne Therapeutics: FORZETTO Explained: Understanding the Phase 3 Trial of Zeleciment Rostudirsen (DYNE-251) for Duchenne Watch HERE
When a child is diagnosed with Duchenne muscular dystrophy, one of the most important parts of care is having a healthcare team that understands the disease. Physical therapy plays an essential role in […]
Capricor Therapeutics recently shared an update on the FDA review of their investigational therapy, deramiocel. The company shared that the Biologics License Application (BLA) for deramiocel remains under FDA review […]
Watch our webinar (in English and Spanish) with helpful insights on who is eligible, common school supports, transition planning, and how to build a strong partnership with your child’s school. […]
ASK ME ANYTHING: Endocrine & Bone Health for People with DBMD Dr. David Weber, Pediatric Endocrinologist at Children’s Hospital of Philadelphia (CHOP) Recorded: Wednesday, August 5, 2026 Watch HERE
Solid Biosciences has provided an update to the community about SGT-003, their AAV-delivered microdystrophin gene therapy. In the Phase 1/2 INSPIRE DUCHENNE clinical trial, 53 boys have been dosed, ranging […]
Capricor Therapeutics has shared that the US FDA will hold an Advisory Committee meeting on July 29, 2026, to discuss the company’s Biological License Application (BLA) seeking approval of deramiocel, […]
Exon 44 Skipping CureDuchenne was an early funder of Avidity Biosciences, which is now a Novartis company. We are pleased to share that they have submitted a BLA to the […]
REGENXBIO has completed dosing in the confirmatory study of RGX-202, an AAV-delivered microdystrophin gene therapy for the treatment of Duchenne. The company is on track to initiate a Biologics License […]
