PBGM01 clinical trials

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

This study is testing a new gene therapy, PBGM01, for children with GM1 gangliosidosis (types 1 and 2a). It aims to see if the treatment is safe and effective in delivering a working gene to help the body fix the underlying problem.