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Interim Safety, Biomarker, and Efficacy Data from Imagine-1: A Phase 1/2 Open-label, Multicenter Study to Assess the Safety, Tolerability, and Efficacy of a Single Dose, ICM Administration of PBGM01 in Subjects with Type I (early Onset) and 10. Type IIa (late Onset) Infantile GM1 Gangliosidosis (GM1)

David A. Weinstein,Caroline A. Hastings,Debra-Lynn Day-Salvatore,Can Ficicioglu,Chester B. Whitley,Michal Inbar-Feigenberg,Genevieve Bernard,Roberto Giugliani,Julien Baruteau,Fatih S. Ezgu,Jeanine R. Jarnes,Yan G. Ni, Pruthvi Nagilla,Victoria L. Ballard,Thomas F. Haws,Michael H. Gelb,Mark S. Forman

MOLECULAR THERAPY（2022）

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