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)
- Resource Type
- Source
- Molecular Genetics and Metabolism. 138:107169
- Subject
Endocrinology Endocrinology, Diabetes and Metabolism Genetics Molecular Biology Biochemistry - Language
- ISSN
- 1096-7192