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Updated interim safety, biomarker, and efficacy data from I..:
Jarnes, Jeanine R.
;
Hastings, Caroline A.
;
Ficicioglu, Can H.
...
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107169 , 2023
Link:
https://doi.org/10.1016/j.ymgme.2022.107169
RT Journal T1
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)
UL https://suche.suub.uni-bremen.de/peid=cr-10.1016_j.ymgme.2022.107169&Exemplar=1&LAN=DE A1 Jarnes, Jeanine R. A1 Hastings, Caroline A. A1 Ficicioglu, Can H. A1 Day-Salvatore, Debra-Lynn A1 Giugliani, Roberto A1 Baruteau, Julien A1 Whitley, Chester B. A1 Inbar-Feigenberg, Michal A1 Bernard, Geneviève A1 Ezgü, Fatih S. A1 Ni, Yan G. A1 Miller, Michelle A1 Gelb, Michael H. A1 Nagilla, Pruthvi A1 Johnstone, Rose A1 Elsasser, Patricia A1 Cunningham, Elizabeth A1 Ballard, Victoria A1 Haws, Thomas F. A1 Forman, Mark S. A1 Weinstein, David A. PB Elsevier BV YR 2023 SN 1096-7192 JF Molecular Genetics and Metabolism VO 138 IS 2 SP 107169 LK http://dx.doi.org/https://doi.org/10.1016/j.ymgme.2022.107169 DO https://doi.org/10.1016/j.ymgme.2022.107169 SF ELIB - SuUB Bremen
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