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 […]
FDA Will Review Dyne Application for Skipping Exon 51 CureDuchenne was an early funder of Dyne Therapeutics, and we are pleased to share that the FDA has accepted the BLA […]
Italfarmaco reported new data from the Phase 3 EPIDYS trial, showing that quantitative MRI measures of muscle mass and fat fraction were significantly different between the treatment and placebo groups. […]
Proposed text: CureDuchenne was an early funder of Sarepta Therapeutics, and we are pleased to share that the US FDA has accepted supplemental New Drug Applications (sNDAs) for AMONDYS 45 […]
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 […]
