Ultragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)
Ultragenyx Pharmaceutical has announced that the European Medicines Agency has validated its Marketing Authorization Application for rebisufligene etisparvovec, an investigational gene therapy for treating Mucopolysaccharidosis type IIIA (Sanfilippo syndrome Type A). Validation allows EMA to begin formal scientific review, marking a significant reg…