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FDA accepts Sarepta’s application to convert AMONDYS 45 and VYONDYS 53 from accelerated to full approval
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 […]
Update from Solid on microdystrophin gene therapy program
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 […]
FDA will hold Advisory Committee meeting on July 29th to review deramiocel for treatment of Duchenne
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, […]
Avidity submits Biological License Application (BLA) to the FDA, seeking approval for exon 44-skipping “del zota”
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 completes dosing in Duchenne gene therapy confirmation study; planning BLA submission to FDA in Q3 2026
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 […]
cTAP Announces Novel Prognostic Score Developed for Duchenne Muscular Dystrophy Patients Offers Improved Prediction of Loss of Ambulation
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 […]
CureDuchenne Invests in SonoThera to Advance Nonviral Gene Therapy Platform for Duchenne Muscular Dystrophy
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 […]
Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027
Read Avidity’s Community Letter: