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

Published 2020 View Full Article

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Title
Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial
Authors
Keywords
Metachromatic leukodystrophy, Lysosomal storage disease, Enzyme replacement therapy, Intrathecal, Arylsulfatase A
Journal
MOLECULAR GENETICS AND METABOLISM
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-07-16
DOI
10.1016/j.ymgme.2020.07.002

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