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A conceptual disease model for adult Pompe disease

BACKGROUND: Studies in orphan diseases are, by nature, confronted with small patient populations, meaning that randomized controlled trials will have limited statistical power. In order to estimate the effectiveness of treatments in orphan diseases and extrapolate effects into the future, alternativ...

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Detalles Bibliográficos
Autores principales:	Kanters, Tim A., Redekop, W. Ken, Rutten-Van Mölken, Maureen P.M.H., Kruijshaar, Michelle E., Güngör, Deniz, van der Ploeg, Ans T., Hakkaart, Leona
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2015
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4570629/ https://www.ncbi.nlm.nih.gov/pubmed/26374742 http://dx.doi.org/10.1186/s13023-015-0334-6

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