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A Prospective, Cross-Sectional Survey Study of the Natural History of Niemann-Pick B Disease
OBJECTIVE: The objective of this study was to characterize the clinical features of patients with Niemann-Pick disease Type B and to identify efficacy endpoints for future clinical trials of enzyme replacement therapy. PATIENTS AND METHODS: Fifty-nine patients who had Niemann-Pick disease Type B, we...
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| Main Authors: | , , , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2008
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2692309/ https://ncbi.nlm.nih.gov/pubmed/18625664 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1542/peds.2007-3016 |
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