Product
PBGM01
1 clinical trial
4 indications
Indication
GM1 gangliosidosisIndication
Type IIndication
Type 2Indication
GLB1 DeficiencyClinical 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