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, Matthias; Berger, Kenneth I.; Díaz-Manera, Jordi; Dimachkie, Mazen M.; Hamed, Alaa; Riou França, Lionel; Thibault, Nathan; Shukla, Pragya; Ishak, Jack; Caro, J. Jaime (Orphanet journal of rare diseases, 2024)
Keywords: Avalglucosidase alfa; Care and treatment; Clinical trial; Clinical trials; Comparative analysis; Complications and side effects; Diagnosis; Disease; Enzyme replacement therapy; Enzymes; Evaluation; Glycogenosis; Human Genetics; Late-onset Pompe disease; Medical research; Medicine; Medicine & Public Health; Methods; Orphan drugs; Patient outcomes; Pharmacology/Toxicology; Product development; Rare diseases; Ratios; Win ratio; United States; Clinical trial; Avalglucosidase alfa; Enzyme replacement therapy; Late-onset Pompe disease; Win ratio