Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial

Publication date: Available online 16 July 2020Source: Molecular Genetics and MetabolismAuthor(s): Christine Í. Dali, Caroline Sevin, Ingeborg Krägeloh-Mann, Roberto Giugliani, Norio Sakai, James Wu, Margaret Wasilewski
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research