640P Efficacy and safety of EPI-321, an investigational single dose epigenome editing therapy targeting D4Z4 in facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral muscular dystrophy Epigenome
DOI: 10.1016/j.nmd.2024.07.383 Publication Date: 2024-10-04T06:55:56Z