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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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2015
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4570629/
https://ncbi.nlm.nih.gov/pubmed/26374742
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0334-6
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