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

Authors: Matthias Boentert, Kenneth I. Berger, Jordi Díaz-Manera, Mazen M. Dimachkie, Alaa Hamed, Lionel Riou França, Nathan Thibault, Pragya Shukla, Jack Ishak, J. Jaime Caro

Source: Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-8 (2024)

Subject Terms: Late-onset Pompe disease, Clinical trial, Win ratio, Enzyme replacement therapy, Avalglucosidase alfa, Medicine

File Description: electronic resource

Relation: https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/e7b737f3c80f41c2a3616eefc9531926

Cost‐Effectiveness Analysis of Empagliflozin for Treatment of Patients With Heart Failure With Reduced Ejection Fraction in the United States

Authors: Odette S. Reifsnider, Ali Tafazzoli, Stephan Linden, Jack Ishak, Pal Rakonczai, Matthew Stargardter, Effie Kuti

Source: Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease, Vol 13, Iss 4 (2024)

Subject Terms: cost‐effectiveness, empagliflozin, heart failure, sodium–glucose co‐transporter 2 inhibitor, United States, Diseases of the circulatory (Cardiovascular) system, RC666-701

File Description: electronic resource

Relation: https://doaj.org/toc/2047-9980

Access URL: https://doaj.org/article/8be347aa15bf47fe965fc52f141023d4

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