AXO-AAV-GM2 Gene Therapy for Infantile- and Juvenile-onset GM2 Gangliosidosis: Preliminary Results from an Ongoing Phase 1/2 Trial

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

MOLECULAR THERAPY, 2022; 30 (5): 6

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