Infantile Gm2 Gangliosidosis
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CLINICAL_TRIALS trial_result · 2023-05-09Clinical Trial Update: First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis [Phase 1, Phase 2]
First-in-human study of TSHA-101 gene therapy for infantile GM2 gangliosidosis is underway in Phase 1/2.