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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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Dades bibliogràfiques
Publicat a:Nat Rev Methods Primers
Autors principals: Pizzamiglio, Chiara, Vernon, Hilary J., Hanna, Michael G., Pitceathly, Robert D. S.
Format: Artigo
Idioma:Inglês
Publicat: 2022
Matèries:
Accés en línia: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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