BETA-GALACTOSIDASE-1 (GLB1) DEFICIENCY

Clinical trials for BETA-GALACTOSIDASE-1 (GLB1) DEFICIENCY explained in plain language.

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One-Time gene therapy aims to halt rare, fatal brain disease in children

Disease control Ongoing

This study tests a single dose of PBGM01, a gene therapy delivered directly into the fluid around the brain and spinal cord, in children with GM1 gangliosidosis. The therapy uses a harmless virus to carry a working copy of the GLB1 gene, which is missing or faulty in these patien…

Matched conditions: BETA-GALACTOSIDASE-1 (GLB1) DEFICIENCY

Phase: PHASE1, PHASE2 • Sponsor: Gemma Biotherapeutics • Aim: Disease control

Last updated Jul 02, 2026 00:00 UTC

BETA-GALACTOSIDASE-1 (GLB1) DEFICIENCY

Get alerted when new BETA-GALACTOSIDASE-1 (GLB1) DEFICIENCY trials appear

One-Time gene therapy aims to halt rare, fatal brain disease in children