Sarepta Therapeutics Shares Surge on Rare Disease Drug Data
Sarepta Therapeutics shares rose 20% after releasing early clinical data for two rare muscle-wasting disease candidates, SRP-1001 and SRP-1003.
Gene Therapy
2 articles tagged "Gene Therapy"
FDA Approves Kresladi Gene Therapy for Rare LAD-1 Disease
The FDA has approved Rocket Pharma's Kresladi for LAD-1, a rare immunodeficiency affecting roughly 25 US patients annually, marking a gene therapy milestone.
By Robert Kim