SA26 Win Ratio Analyses of Multiple Endpoints in Rare Disease Trials: A Case-Study Based on a Trial of Avaglucosidase Alfa in Late-Onset Pompe Disease (LOPD)

Abstract

Authors

KJ Ishak JJ Caro A Hamed L Riou-Franca N Thibault P Shukla KI Berger J Diaz-Manera MM Dimachkie M Boentert