Details van artikel 259 van 350 gevonden artikelen
Phase 1/2 open-label, multi-center study to assess the safety, tolerability and efficacy of a single dose of PBGM01 delivered into the cisterna magna of subjects with type 1 (early onset) and type 2a (late onset) infantile GM1 gangliosidosis
Titel:
Phase 1/2 open-label, multi-center study to assess the safety, tolerability and efficacy of a single dose of PBGM01 delivered into the cisterna magna of subjects with type 1 (early onset) and type 2a (late onset) infantile GM1 gangliosidosis
Auteur:
Jarnes, Jeanine R. Day-Salvatore, Debra L. Ficicioglu, Can Hastings, Caroline Bernard, Genevieve Baruteau, Julien Ezgu, Fatih Giugliani, Roberto Inbar-Feigenberg, Michal Whitley, Chester B. Ni, Yan Ballard, Victoria L. Haws, Thomas F. Weinstein, David A.
Verschenen in:
Molecular genetics and metabolism
Paginering:
Jaargang 135 () nr. 2 pagina's S62-S63
Jaar:
2022
Inhoud:
Uitgever:
Published by Elsevier B.V.
Bronbestand:
Elektronische Wetenschappelijke Tijdschriften
Details van artikel 259 van 350 gevonden artikelen