Ongoing Sanfilippo Syndrome Type A (MPS IIIA) Gene Therapy Clinical Trial Enrolling Patients

Abeona Therapeutics, Inc. is enrolling patients in a Phase I/II non-randomized, open-label clinical trial studying a one-time gene therapy for Sanfilippo syndrome type A (MPS IIIA). They have entitled this study ‘The Transpher A Study.’ Caregivers interested in enrolling their children can visit to take an eligibility survey and learn more about the study. Additional details can be found at under study number NCT02716246 or by contacting