Composite endpoints, including patient reported outcomes, in rare diseases

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Authors: Johan Verbeeck; Maya Dirani; Johann W. Bauer; Ralf-Dieter Hilgers; Geert Molenberghs; Rima Nabbout

Journal title: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-11 (2023)

Journal number: 8

Journal publisher: BioMed Central

Published year: 2023

DOI identifier: 10.1186/s13023-023-02819-x

ISSN: 1750-1172