Broad FDA Approval of Gene Therapy Expands Treatment Choices for Duchenne Muscular Dystrophy: Debra Miller & Michael Kelly, PhD
AFFINITY DUCHENNE Trial Expands, Suvecaltamide Fails Phase 2b Study of Essential Tremor, PTC518 Shows Dose-Dependent Huntingtin Lowering
ABBV-951 Gets Another CRL, Pitolisant Approved for Pediatric Narcolepsy, FDA Clears Efgartigimod for CIDP