Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial
Titel:
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial
Auteur:
Schoser, Benedikt Roberts, Mark Byrne, Barry J Sitaraman, Sheela Jiang, Hai Laforêt, Pascal Toscano, Antonio Castelli, Jeff Díaz-Manera, Jordi Goldman, Mitchell van der Ploeg, Ans T Bratkovic, Drago Kuchipudi, Srilakshmi Mozaffar, Tahseen Kishnani, Priya S