Items where Author is "Boentert, Matthias"

Boentert, Matthias, Berger, Kenneth I., Díaz-Manera, Jordi, Dimachkie, Mazen M., Hamed, Alaa, Riou França, Lionel, Thibault, Nathan, Shukla, Pragya, Ishak, Jack and Caro, J. Jaime (2024) 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. Orphanet Journal of Rare Diseases, 19 (1). ISSN 1750-1172