Pharmacokinetic Modeling of Intrathecally Administered Recombinant Human Arylsulfatase A (TAK‐611) in Children With Metachromatic Leukodystrophy

Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by deficient arylsulfatase A (ASA) activity, which leads to neuronal sulfatide accumulation and motor and cognitive deterioration. Intrathecal delivery of a recombinant human ASA (TAK‐611, formerly SHP611) is under development...

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Detalles Bibliográficos
Autores principales: Troy, Steven, Wasilewski, Margaret, Beusmans, Jack, Godfrey, C.J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7325319/
https://www.ncbi.nlm.nih.gov/pubmed/31868225
http://dx.doi.org/10.1002/cpt.1752

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7325319/
https://www.ncbi.nlm.nih.gov/pubmed/31868225
http://dx.doi.org/10.1002/cpt.1752

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