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Designing clinical trials for rare diseases: unique challenges and opportunities

Orphan drug development is a rapidly expanding field. Nevertheless, clinical trials for rare diseases can present inherent challenges. Optimal study design and partnerships between academia and industry are therefore required for the successful development, delivery and clinical approval of effectiv...

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Detalhes bibliográficos
Publicado no:Nat Rev Methods Primers
Main Authors: Pizzamiglio, Chiara, Vernon, Hilary J., Hanna, Michael G., Pitceathly, Robert D. S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2022
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7613711/
https://ncbi.nlm.nih.gov/pubmed/36254116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s43586-022-00100-2
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