Indication
MPS I
4 clinical trials
4 products
Clinical trial
A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)Status: Terminated, Estimated PCD: 2021-11-03
Product
SB-318Clinical trial
A Phase 1/2 Open-Label, Sequential Dose-Escalation, Safety, Tolerability and Efficacy Study of SIG-005 in Adult Patients With Mucopolysaccharidoses 1 (MPS-1)Status: Withdrawn, Estimated PCD: 2022-12-13
Product
SIG-005Clinical trial
Longitudinal Study of Neurodegenerative DisordersStatus: Recruiting, Estimated PCD: 2030-01-01
Clinical trial
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs).Status: Recruiting, Estimated PCD: 2031-07-01
Product
Aldurazyme