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GM1 Gangliosidosis, Type 2 clinical trials at UCSF

1 research study open to eligible people

Showing trials for
  • Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Subjects With GM1 Gangliosidosis

    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

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