AXO-AAV-GM2 Gene Therapy for the Treatment of GM2 Gangliosidosis: Preliminary Results from a Phase 1/2 Trial

Eichler, F; Flotte, T; Andonian, H; Cataltepe, O; Artinian, R; Nagy, A; Thorp, B; Jameson, J; Sheehan, M; Vaughn, T; Valencia, D; De Boever, E

ANNALS OF NEUROLOGY, 2022; 92 (): S169

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