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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...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2020
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| Materias: | |
| 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 |