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.
ESTEVE-SANF-201
Phase 2 other active
Quick answer
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. for Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) is a Phase 2 program (other) at Esteve Pharmaceuticals with 1 ClinicalTrials.gov record(s).
Program details
- Company
- Esteve Pharmaceuticals
- Indication
- Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
- Phase
- Phase 2
- Modality
- other
- Status
- active