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Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease.
Boentert M, Berger KI, Díaz-Manera J, Dimachkie MM, Hamed A, Riou França L, Thibault N, Shukla P, Ishak J, Caro JJ. Boentert M, et al. Among authors: shukla p. Orphanet J Rare Dis. 2024 Jan 12;19(1):14. doi: 10.1186/s13023-023-02974-1. Orphanet J Rare Dis. 2024. PMID: 38216959 Free PMC article.
1,782 results