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 […]
As a founding partner and initial funder of the Collaborative Trajectory Analysis Project (cTAP), CureDuchenne is proud to support an important advancement for the Duchenne community: a novel prognostic score that helps […]
SonoThera press release CureDuchenne is pleased to announce that it has invested in SonoThera, supporting the advancement of its targeted, ultrasound-mediated gene delivery technology focused on the delivery of full-length […]
Read Avidity’s Community Letter:
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 […]
The neuromuscular disease community received encouraging news this week as Edgewise Therapeutics announced the sale of sevasemten and its Becker and Duchenne muscular dystrophy programs to Servier, a global pharmaceutical company with […]
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 […]
