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TrialThe New England journal of medicine2026

AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.

Connor J Lewis et al.PubMed ↗Full text ↗Publisher ↗

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3 papers cite it

2026
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The trial

Full record →Abstract, authors, funding and every citing paper · PMID 41665410