Back to Search
Start Over
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)
- Source :
- Molecular Genetics and Metabolism. 138:107169
- Publication Year :
- 2023
- Publisher :
- Elsevier BV, 2023.
Details
- ISSN :
- 10967192
- Volume :
- 138
- Database :
- OpenAIRE
- Journal :
- Molecular Genetics and Metabolism
- Accession number :
- edsair.doi...........2595e1d8f08ff57da247d7806faa3b67