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Linear Clinical Progression, Independent of Age of Onset, in Niemann-Pick Disease, type C
Niemann-Pick Disease, type C is a neurodegenerative, lysosomal storage disorder with a broad clinical spectrum and a variable age of onset. The absence of a universally accepted clinical outcome measure is an impediment to the design of a therapeutic trial for NPC. Thus, we developed a clinical seve...
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| Main Authors: | , , , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2010
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2798912/ https://ncbi.nlm.nih.gov/pubmed/19415691 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajmg.b.30969 |
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