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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Bibliografski detalji
Izdano u:Orphanet J Rare Dis
Glavni autori: 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
Jezik:Inglês
Izdano: BioMed Central 2015
Teme:
Research
Online pristup: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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