Efficacy and safety of avalglucosidase alfa in participants with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

Authors :: Priya S. Kishnani
Jordi Díaz-Manera
Hani Kushlaf
Shafeeq Ladha
Tahseen Mozaffar
Volker Straub
Antonio Toscano
Ans T. van der Ploeg
Paula R. Clemens
John W. Day
Sergey Illarioshkin
Mark E. Roberts
Shahram Attarian
Gerson Carvalho
Sevim Erdem-Özdamar
Ozlem Goker-Alpan
Anna Kostera-Pruszczyk
Kristina An Haack
Olivier Huynh-Ba
Swathi Tammireddy
Nathan Thibault
Tianyue Zhou
Mazen M. Dimachkie
Benedikt Schoser
Source :: Molecular Genetics and Metabolism. 138:107185
Publication Year :: 2023
Publisher :: Elsevier BV, 2023.

Subjects :: Endocrinology
Endocrinology, Diabetes and Metabolism
Genetics
Molecular Biology
Biochemistry

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