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Sio Gene Therapies Announces CSF Reductions in GM1 Ganglioside from Clinical Trial of AXO-AAV-GM1 Gene Therapy

- GM1 ganglioside in CSF reduced in 4 out of 5 children treated with the lowest dose at 6 months follow-up- Direct evidence of biochemical effect in the CNS suggests intravenous gene therapy may address both the systemic and neurological manifestations o