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
| Autor: | 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. |
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| Zdroj: | In Molecular Genetics and Metabolism February 2022 135(2):S62-S63 |
| Databáze: | ScienceDirect |
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