REGENXBIO Completes RGX-202 Confirmatory Study for Duchenne Muscular Dystrophy
REGENXBIO has announced the completion of patient dosing in the confirmatory study of RGX-202, its investigational gene therapy for Duchenne Muscular Dystrophy (DMD). This...
GEn1E Lifesciences and GEn-1123: A New Precision Medicine Approach for Duchenne Muscular Dystrophy (DMD)
The U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to GEn-1123, an investigational...
RGX-202 Duchenne Gene Therapy Shows NSAA Improvement and Microdystrophin Expression — But Important Biomarker Data Missing
REGENXBIO recently announced positive topline results from the Phase 3 portion of the AFFINITY DUCHENNE trial evaluating RGX-202, an investigational gene therapy for Duchenne...
Entrada Therapeutics Announces Phase 1/2 Results for ENTR-601-44 in Duchenne Muscular Dystrophy
Entrada Therapeutics announced early clinical trial results for ENTR-601-44, an experimental treatment for people with Duchenne Muscular Dystrophy (DMD). The therapy is designed for...
European Commission Grants Orphan Drug Designation to SGT-003 for Duchenne Muscular Dystrophy
The European Commission has granted Orphan Drug Designation to SGT-003, a promising gene therapy developed by Solid Biosciences, for the treatment of Duchenne muscular...