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Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditions

BACKGROUND: Randomized controlled trials (RCTs) are the gold standard design of clinical research to assess interventions. However, RCTs cannot always be applied for practical or ethical reasons. To investigate the current practices in rare diseases, we review evaluations of therapeutic intervention...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Unkel, Steffen, Röver, Christian, Stallard, Nigel, Benda, Norbert, Posch, Martin, Zohar, Sarah, Friede, Tim
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2016
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4761188/
https://ncbi.nlm.nih.gov/pubmed/26897367
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-016-0402-6
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