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. […]
The addition of Duchenne muscular dystrophy (DMD) to the Recommended Uniform Screening Panel (RUSP) marks a meaningful step forward in the effort to diagnose Duchenne earlier and connect families to […]
Terapia Génica ELEVIDYS: Actualización Comunitaria Más Reciente con Sarepta Therapeutics Watch HERE
Rosalia es una orgullosa mamá de Joe (8), quien vive con Duchenne, y de Sophia (6). Ella misma vive con distrofinopatía, lo que hace que esta misión sea especialmente cercana […]
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 […]
Capricor Therapeutics, which received early funding from CureDuchenne, said the FDA has requested the full clinical study report from their latest clinical trial, the HOPE-3 trial. Positive topline results from […]
