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Designing clinical trials for rare diseases: unique challenges and opportunities

Pizzamiglio, C; Vernon, HJ; Hanna, MG; Pitceathly, RDS; (2022) Designing clinical trials for rare diseases: unique challenges and opportunities. Nature Reviews Methods Primers , 2 , Article 13. 10.1038/s43586-022-00100-2. Green open access

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Type: Article
Title: Designing clinical trials for rare diseases: unique challenges and opportunities
Open access status: An open access version is available from UCL Discovery
DOI: 10.1038/s43586-022-00100-2
Publisher version: https://doi.org/10.1038/s43586-022-00100-2
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
UCL classification: UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
URI: https://discovery.ucl.ac.uk/id/eprint/10150422
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