Indication
Mucopolysaccharidosis type IIIA
2 clinical trials
3 products
Clinical trial
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIAStatus: Active (not recruiting), Estimated PCD: 2024-06-01
Product
ABO-102Product
Adjuvant ImmunosuppressionClinical trial
A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA DiseaseStatus: Terminated, Estimated PCD: 2022-03-10