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

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-C patie...

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Detalles Bibliográficos
Autores principales: Patterson, Marc C., Mengel, Eugen, Vanier, Marie T., Moneuse, Patrick, Rosenberg, Daniel, Pineda, Mercedes
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7183679/
https://www.ncbi.nlm.nih.gov/pubmed/32334605
http://dx.doi.org/10.1186/s13023-020-01363-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7183679/
https://www.ncbi.nlm.nih.gov/pubmed/32334605
http://dx.doi.org/10.1186/s13023-020-01363-2

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