Capricor Therapeutics announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy […]
Precision BioSciences has shared that the first participant in the FUNCTION_DMD clinical trial has received their investigational gene editing treatment, PBGENE-DMD. PBGENE-DMD delivers ARCUS nucleases via AAV9 to cut out […]
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 […]
As an early funder of Entrada Therapeutics, we are pleased to share their latest newsletter to the Duchenne community. This update includes news that after review of all the data […]
CureDuchenne was an early funder of Dyne Therapeutics, which today announced they have submitted a BLA to the FDA for z-rostudirsen, for the treatment of individuals with Duchenne amenable to […]
Dyne Therapeutics, which received early funding from CureDuchenne, is initiating a Phase 3 FORZETTO Trial of z-rostudirsen in individuals with Duchenne amenable to skipping exon 51. Dyne is on track […]
