Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study

OBJECTIVE: Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease caused by deficient activity of arylsulfatase A (ASA), resulting in severe motor and cognitive dysfunction. This phase 1/2 study evaluated the safety and efficacy of intravenous (IV) recombinant human A...

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Bibliografiske detaljer
Udgivet i:Ann Clin Transl Neurol
Main Authors: í Dali, Christine, Groeschel, Samuel, Moldovan, Mihai, Farah, Mohamed H., Krägeloh‐Mann, Ingeborg, Wasilewski, Margaret, Li, Jing, Barton, Norman, Krarup, Christian
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7818087/
https://ncbi.nlm.nih.gov/pubmed/33332761
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51254
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