Indication

GLB1 Deficiency

1 clinical trial

1 product

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 Gangliosidosis

Status: Active (not recruiting), Estimated PCD: 2026-02-01

Product