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. 2016 Nov;15(11):731-732.
doi: 10.1038/nrd.2016.133. Epub 2016 Jul 22.

Patients as key partners in rare disease drug development

Max G Bronstein  1 , Emil D Kakkis  2   3
Affiliations

Patients as key partners in rare disease drug development

Max G Bronstein et al. Nat Rev Drug Discov. 2016 Nov.

Abstract

Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit-risk profile of potential therapies.

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