Evolution of structural neuroimaging biomarkers in a series of adult patients with Niemann-Pick type C under treatment

BACKGROUND: Niemann-Pick type C (NPC) disease is a lysosomal storage disorder characterized by a wide clinical spectrum and non-specific conventional magnetic resonance imaging (MRI) signs. As substrate reduction therapy with miglustat is now used in almost all patients, its efficacy and the course...

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Detalles Bibliográficos
Autores principales: Masingue, Marion, Adanyeguh, Isaac, Nadjar, Yann, Sedel, Frédéric, Galanaud, Damien, Mochel, Fanny
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5289046/
https://www.ncbi.nlm.nih.gov/pubmed/28148276
http://dx.doi.org/10.1186/s13023-017-0579-3

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