As an early investor in Dyne Therapeutics, CureDuchenne is happy to share that new analyses out to 24 months showed improvement in heart and lung function with z-rostudirsen compared to […]
Roche has decided to stop recruitment for their Phase 2 SHIELD DMD study of satralizumab in Duchenne, citing the decision was not due to any new efficacy or safety issues, but rather […]
We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for Translarna™ (ataluren) for the treatment of nonsense mutation DMD after feedback from the FDA that the […]
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Precision BioSciences announced that the U.S. FDA has cleared its Investigational New Drug (IND) application for a gene editing therapy for Duchenne muscular dystrophy. This clearance allows the company to move […]
CureDuchenne Invests in Satellos to Advance a Mutation-Independent Duchenne TherapyLink to press release CureDuchenne is pleased to announce that it has participated in the Satellos Bioscience public offering, supporting the […]
Solid Biosciences shared that they have aligned with the US FDA on the overall design of a Phase 3 randomized, double-blind, placebo-controlled trial for SGT-003, a micro-dystrophin gene therapy for Duchenne. […]
Avidity Biosciences has released answers to frequently asked questions about their Managed Access Program (MAP) for individuals with Duchenne who are amenable to exon 44 skipping and meet clinical criteria. […]
Solid Biosciences reports that 33 children have now been dosed in their SGT-003 gene therapy trial for Duchenne. So far, the treatment has been generally well tolerated with signs of improved muscle […]
