Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry

Abstract Background Niemann-Pick disease Type C (NP-C) is a rare, progressive neurodegenerative disorder characterized by progressive neurodegeneration and premature death. We report data at closure of the NPC Registry that describes the natural history, disease course and treatment experience of NP...

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Principais autores:	Marc C. Patterson, Eugen Mengel, Marie T. Vanier, Patrick Moneuse, Daniel Rosenberg, Mercedes Pineda
Formato:	Artigo
Idioma:	Inglês
Publicado em:	BMC 2020-04-01
coleção:	Orphanet Journal of Rare Diseases
Assuntos:	Niemann-Pick disease type C Miglustat NPC disease registry Treatment evaluation Neurological symptoms Safety
Acesso em linha:	http://link.springer.com/article/10.1186/s13023-020-01363-2
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