Gm1 Gangliosidosis Type
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial [Phase 1]
A Phase 1 prenatal gene therapy trial using an AAV9 vector delivering human beta-galactosidase for GM1 gangliosidosis type I and type II; cu…
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CLINICAL_TRIALS research · 2025-05-21Clinical Trial Update: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis [Phase 1, Phase 2]
Gemma Biotherapeutics reports an ongoing Phase 1/2 trial evaluating PBGM01 for safety, tolerability, and efficacy in pediatric GM1 gangliosi…