Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: a 5-year update from the UK clinical database

BACKGROUND: Niemann-Pick disease type C (NP-C) is a rare neurovisceral lipid storage disorder characterised by progressive, disabling neurological symptoms and premature death in most patients. During the last decade, national cohort studies have accrued a great deal of data on the symptomatology an...

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Detalles Bibliográficos
Autores principales: Imrie, Jackie, Heptinstall, Lesley, Knight, Stephen, Strong, Kate
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4678528/
https://www.ncbi.nlm.nih.gov/pubmed/26666848
http://dx.doi.org/10.1186/s12883-015-0511-1

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