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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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Pubblicato in:Nat Rev Methods Primers
Autori principali: Pizzamiglio, Chiara, Vernon, Hilary J., Hanna, Michael G., Pitceathly, Robert D. S.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2022
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Accesso online: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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