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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Dades bibliogràfiques
Publicat a:Orphanet J Rare Dis
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2015
Matèries:
Research
Accés en línia: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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