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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
81 auth.
B. Schoser,
M. Roberts,
B. Byrne,
S. Sitaraman,
Hai Jiang,
P. Laforêt,
A. Toscano,
J. Castelli,
J. Díaz-Manera,
M. Goldman,
A. T. Ploeg,
D. Bratkovic,
Srilakshmi Kuchipudi,
T. Mozaffar,
P. Kishnani,
...
Á. Sebők,
A. Pestronk,
A. Dominović-Kovačević,
Aneal Khan,
B. Koritnik,
C. Tard,
C. Lindberg,
C. Quinn,
Crystal Eldridge,
C. Bodkin,
David Reyes-Leiva,
D. Hughes,
Ela Stefanescu,
E. Salort-Campana,
E. Butler,
F. Bouhour,
G. Kim,
G. Papadimas,
G. Parenti,
H. Bartosik-Psujek,
H. Kushlaf,
Hashiguchi Akihiro,
H. Lau,
Helio F. Pedro,
H. Andersen,
H. Amartino,
H. Shiraishi,
Hiroshi Kobayashi,
Ivaylo Tarnev,
J. Vengoechea,
Jenny Avelar,
Jin-Hong Shin,
Jonathan Cauci,
J. Alonso-Pérez,
J. Janszky,
J. Berthy,
Kornblum Cornelia,
K. Gutschmidt,
K. Claeys,
M. Molnár,
M. Wencel,
M. Tarnopolsky,
M. Dimachkie,
M. Tchan,
M. Freimer,
N. Longo,
Núria Vidal-Fernandez,
O. Musumeci,
O. Goker-Alpan,
P. Deegan,
P. Clemens,
R. Roxburgh,
R. Henderson,
R. Hopkin,
S. Sacconi,
S. Fecarotta,
S. Attarian,
S. Wenninger,
Stephanie M Dearmey,
T. Hiwot,
T. Burrow,
T. Ruck,
Tomohiko Sawada,
Vescei Laszlo,
W. Löscher,
Y. Chien
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5 |
2021 |
5
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