Intrathecal enzyme replacement therapy reverses cognitive decline in mucopolysaccharidosis type I

Mucopolysaccharidosis type I (MPS I) is an inherited lysosomal storage disease that seriously affects the brain. Severity of neurocognitive symptoms in attenuated MPS subtype (MPS IA) broadly varies partially, due to restricted permeability of blood‐brain barrier (BBB) which limits treatment effects...

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Detalhes bibliográficos
Publicado no:Am J Med Genet A
Main Authors: Nestrasil, Igor, Shapiro, Elsa, Svatkova, Alena, Dickson, Patricia, Chen, Agnes, Wakumoto, Amy, Ahmed, Alia, Stehel, Edward, McNeil, Sarah, Gravance, Curtis, Maher, Elizabeth
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2017
Assuntos:
Clinical Reports
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5367919/
https://ncbi.nlm.nih.gov/pubmed/28211988
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.a.38073
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