Precision BioSciences has shared that the first participant in the FUNCTION_DMD clinical trial has received their investigational gene editing treatment, PBGENE-DMD. PBGENE-DMD delivers ARCUS nucleases via AAV9 to cut out […]
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 […]
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 […]
Solid Biosciences has announced the first participant has been dosed in the Phase 3 IMPACT DUCHENNE trial evaluating SGT-003. The global study is now underway, with active sites in Australia […]
Edgewise Therapeutics, a CureDuchenne funded company, today announced positive long-term Sevasemten data that demonstrated sustained functional stabilization in Becker Muscular Dystrophy (BMD) patients through 3.5 years of treatment. This result […]
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 […]
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 […]
REGENXBIO announced new, positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial. They expect to share pivotal topline data […]
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