Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

BACKGROUND: Niemann-Pick disease type C (NP-C) is a rare neurovisceral disease characterised by progressive neurological degeneration, where the rate of neurological disease progression varies depending on age at neurological onset. We report longitudinal data on functional disease progression and s...

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Detalles Bibliográficos
Autores principales: Patterson, Marc C, Mengel, Eugen, Vanier, Marie T, Schwierin, Barbara, Muller, Audrey, Cornelisse, Peter, Pineda, Mercè
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4462071/
https://www.ncbi.nlm.nih.gov/pubmed/26017010
http://dx.doi.org/10.1186/s13023-015-0284-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4462071/
https://www.ncbi.nlm.nih.gov/pubmed/26017010
http://dx.doi.org/10.1186/s13023-015-0284-z

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