A phase 1/2 open-label, multicenter, dose ranging and confirmatory study to assess the safety, tolerability and efficacy of PBKR03 administered to pediatric subjects with early infantile Krabbe disease (globoid cell leukodystrophy; GALax-C)

Category Primary study
JournalMolecular Genetics and Metabolism
Year 2022
This article has no abstract
Epistemonikos ID: 97a391f1da562eeb2abb69f331e659d09980619f
First added on: Feb 13, 2025