Updated 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, intra-cisterna magna (ICM) administration of PBGM01 in subjects with type I (early onset) and type IIA (late onset) infantile GM1 gangliosidosis (GM1)

Jeanine R Jarnes,Michelle Miller,Pruthvi Nagilla,Elizabeth Cunningham,Thomas F Haws,Roberto Giugliani,Debra-Lynn Day-Salvatore,Mark S Forman,Michael H Gelb,Julien Baruteau,David A Weinstein,Patricia Elsasser,Chester B Whitley,Geneviève Bernard,Rose M Johnstone ,Michal Inbar‐Feigenberg ,Caroline Hastings ,Victoria L T Ballard ,Fatih Süheyl Ezgü ,Can Fıçıcıoğlu ,Ni Yan

doi:10.1016/j.ymgme.2022.107169

Updated 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, intra-cisterna magna (ICM) administration of PBGM01 in subjects with type I (early onset) and type IIA (late onset) infantile GM1 gangliosidosis (GM1)

Jeanine R Jarnes, Michelle Miller + Show 19 more

https://doi.org/10.1016/j.ymgme.2022.107169

Copy DOI

Journal: Biochemical and Molecular Medicine

Publication Date: Feb 1, 2023

#Infantile GM1 Gangliosidosis #Intra-cisterna Magna + Show 8 more

Abstract
Full-Text PDF
Similar Papers

Abstract

Updated 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, intra-cisterna magna (ICM) administration of PBGM01 in subjects with type I (early onset) and type IIA (late onset) infantile GM1 gangliosidosis (GM1)

Full Text