Disease and patient characteristics in NP-C patients: findings from an international disease registry

BACKGROUND: Niemann-Pick disease type C (NP-C) is a rare neurovisceral disease characterized by progressive neurodegeneration and premature death. We report data recorded at enrolment in an ongoing international NP-C registry initiated in September 2009 to describe disease natural history, clinical...

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Detalles Bibliográficos
Autores principales: Patterson, Marc C, Mengel, Eugen, Wijburg, Frits A, Muller, Audrey, Schwierin, Barbara, Drevon, Harir, Vanier, Marie T, Pineda, Mercé
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2013
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3558399/
https://www.ncbi.nlm.nih.gov/pubmed/23324478
http://dx.doi.org/10.1186/1750-1172-8-12

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3558399/
https://www.ncbi.nlm.nih.gov/pubmed/23324478
http://dx.doi.org/10.1186/1750-1172-8-12

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