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...
New Hope for Duchenne: Ryoncil Clinical Trial Begins
A new breakthrough may change the future of Duchenne muscular dystrophy. Ryoncil (remestemcel-L-rknd), a novel cell therapy, has received FDA clearance for a key...
Regenxbio Reports New Positive Interim Data From Phase 1/2 Affinity Duchenne Trial Of Rgx-202
REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy....