104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)

Schoser, Benedikt ; Kishnani, Priya S. ; Bratkovic, Drago ; Byrne, Barry J. ; Claeys, Kristl G. ; Díaz-Manera, Jordi ; Laforêt, Pascal ; Roberts, Mark ; Toscano, Antonio ; van der Ploeg, Ans T. ; Castelli, Jeff ; Goldman, Mitchell ; Holdbrook, Fred ; Sitaraman Das, Sheela ; Wasfi, Yasmine ; Mozaffar, Tahseen

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Berlin/Heidelberg: Springer Berlin Heidelberg

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