This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. […]
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– ENTR-601-44 was well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events and no clinically significant changes or trends noted in vital signs, ECGs, physical exams or […]
REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory […]
Cranbury Pharmaceuticals Receives U.S. FDA Approval for First Generic Version of Emflaza® Oral Suspension (deflazacort) for Duchenne.
Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy.
Read the full news release HERE Capricor Therapeutics, which received early funding from CureDuchenne, has announced positive 3-year results from the HOPE-2 open-label extension study. Individuals receiving CAP-1002 continue to […]
As a funder of hC Bioscience, we’re happy to have them announcing their tRNA-based approach for targeting nonsense mutations at our annual Futures conference.
As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates in their next-generation exon skipping program for Duchenne amenable to skipping exon 51. In their […]
