Sanfilippo Syndrome Type A(MPS IIIA) Clinical Trials Clinical Trials Recessive Dystrophic Epidermolysis Bullosa (RDEB) Sanfilippo syndrome type A (MPS IIIA) Transpher A Study ABT-003 Sanfilippo syndrome type B (MPS IIIB) An open-label, dose-escalation clinical trial assessing the safety of one-time gene therapy ABO-102 for younger, higher-functioning patients with MPS IIIA. View Study An open-label clinical trial assessing the safety of one-time gene therapy ABO-102 for patients with middle and advanced phases of MPS IIIA. View Study
