학술논문
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)
Document Type
Article
Author
Jarnes, Jeanine R.; Hastings, Caroline A.; Ficicioglu, Can H.; Day-Salvatore, Debra-Lynn; Giugliani, Roberto; Baruteau, Julien; Whitley, Chester B.; Inbar-Feigenberg, Michal; Bernard, Geneviève; Ezgü, Fatih S.; Ni, Yan G.; Miller, Michelle; Gelb, Michael H.; Nagilla, Pruthvi; Johnstone, Rose; Elsasser, Patricia; Cunningham, Elizabeth; Ballard, Victoria; Haws, Thomas F.; Forman, Mark S.
Source
Subject
*BIOMARKERS
*SAFETY
*
Language
ISSN
1096-7192