CureDuchenne was founded 25 years ago, and during that time we have witnessed and participated in the approval of multiple FDA-approved therapies, dozens of clinical trials, and an expanding pipeline of investigational treatments. But there is still no cure for […]
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 […]
CureDuchenne has submitted comments to the U.S. Food and Drug Administration (FDA) on several proposed policies that could affect how new gene therapies and other genetic treatments are developed and reviewed for Duchenne muscular dystrophy, Becker muscular dystrophy, and other rare […]
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 […]
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 […]
By Jason Mast
PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA). The update notes that a limited remaining supply […]
What Sarepta Therapeutics’ sNDA submission means for Duchenne Sarepta Therapeutics is preparing to submit supplemental new drug applications (sNDAs) to the FDA for converting the accelerated approvals of AMONDYS 45 […]
