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Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) research hub

1 indexed studies · 0 currently recruiting

ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients with MPSIIIA.
This study is testing a new gene therapy for children with Sanfilippo A syndrome (MPSIIIA). Researchers want to see if it's safe, tolerable, and if it could help improve their condition. It's the first time this treatment is being given to humans.
Spain