GM1 Gangliosidosis, Type 2 clinical trials at UCSF
1 research study open to eligible people
open to eligible people ages 4 months to 36 months
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2-stage design the safety, tolerability and efficacy of this treatment in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis. Results from the Type 1 and Type 2a groups will be assessed separately.
Oakland, California and other locations