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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Detalles Bibliográficos
Autores principales: í Dali, Christine, Groeschel, Samuel, Moldovan, Mihai, Farah, Mohamed H., Krägeloh‐Mann, Ingeborg, Wasilewski, Margaret, Li, Jing, Barton, Norman, Krarup, Christian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7818087/
https://www.ncbi.nlm.nih.gov/pubmed/33332761
http://dx.doi.org/10.1002/acn3.51254

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7818087/
https://www.ncbi.nlm.nih.gov/pubmed/33332761
http://dx.doi.org/10.1002/acn3.51254

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