Indication
GM1 gangliosidosis
2 clinical trials
2 products
Clinical trial
Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 GangliosidosisStatus: Active (not recruiting), Estimated PCD: 2026-02-01
Product
PBGM01Clinical trial
An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human β-Galactosidase cDNA for Treatment of GM1 GangliosidosisStatus: Terminated, Estimated PCD: 2023-05-22
Product
LYS-GM101