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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Detalles Bibliográficos
Autores principales: Nestrasil, Igor, Shapiro, Elsa, Svatkova, Alena, Dickson, Patricia, Chen, Agnes, Wakumoto, Amy, Ahmed, Alia, Stehel, Edward, McNeil, Sarah, Gravance, Curtis, Maher, Elizabeth
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Clinical Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5367919/
https://www.ncbi.nlm.nih.gov/pubmed/28211988
http://dx.doi.org/10.1002/ajmg.a.38073

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5367919/
https://www.ncbi.nlm.nih.gov/pubmed/28211988
http://dx.doi.org/10.1002/ajmg.a.38073

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