Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy
Greenberg Barry, Coggins Matthew, Schwartz Jonathan, Rossano Joseph, Taylor Matthew, Colan Steven, Adler Eric, Ricks David, Yarabe Paul, Battiprolu Pavan, Shah Guarav, Patel Kinnari
Gene Therapy for Lysosomal Storage Disorders: From the Bench to the Bedside
Adler Eric, Greenberg Barry