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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial
135 auth.
J. Díaz-Manera,
P. Kishnani,
H. Kushlaf,
S. Ladha,
T. Mozaffar,
V. Straub,
A. Toscano,
A. T. Ploeg,
K. Berger,
P. Clemens,
Y. Chien,
J. Day,
S. Illarioshkin,
M. Roberts,
S. Attarian,
...
J. Borges,
F. Bouhour,
Young-Chul Choi,
S. Erdem-Ozdamar,
O. Goker-Alpan,
A. Kostera-Pruszczyk,
K. Haack,
C. Hug,
O. Huynh-Ba,
Judith Johnson,
N. Thibault,
Tianyue Zhou,
M. Dimachkie,
B. Schoser,
A. Béhin,
M. Boentert,
Gerson Carvalho,
N. Chahin,
J. Charrow,
P. Deegan,
H. D. Tekçe,
F. Duval,
A. Genge,
L. Gutmann,
R. Henderson,
J. Hennermann,
T. Hiwot,
D. Hughes,
A. Karaa,
C. Karam,
A. Kautzky-Willer,
H. Komaki,
P. Laforêt,
N. Longo,
V. Malinová,
R. Maré,
C. Maxit,
E. Mengel,
M. Moggio,
M. Molnár,
T. Mongini,
A. Nadaj-Pakleza,
A. N. Osorio,
J. Noury,
A. Oliveira,
Y. Parman,
Loren D M Pena,
G. Remiche,
M. Sciacco,
P. Shieh,
Cheryl Smith,
T. Stulnig,
F. Taithe,
C. Tard,
M. Tarnopolsky,
M. Vorgerd,
Chet R. Whitley,
P. Young,
J. Alonso-Pérez,
Patricia Altemus,
A-C Aubé-Nathier,
Jenny Avelar,
Carrie Bailey,
C. Bekircan-Kurt,
Jenny Billy,
S. Boschi,
K. E. Brown,
Laura Carrera Garcia,
L. Chase,
Hamilton Cirne,
Loïc Danjoux,
Jean-Baptiste Davion,
Stephanie M Dearmey,
E. Fedotova,
Eve Gandolfo,
Z. Grosz,
D. Guellec,
A. Guettsches,
M. Guglieri,
E. Hatcher,
Sina Helms,
Miriam Hufgard-Leitner,
S. Klyushnikov,
J. Langton,
L. Linková,
N. Mavroudakis,
S. Mazurová,
Madoka Mori,
Louisa Müller-Miny,
O. Musumeci,
C. Nance,
D. Benito,
Robert Neel,
G. Niizawa,
L. Noll,
Erik Ortega,
M. Pasnoor,
V. Pautot,
A. Potulska-Chromik,
A. Pugliese,
C. Questienne,
Margarida Lopes,
David Reyes-Leiva,
M. Riedl,
M. Rugiero,
E. Salort-Campana,
Paulo Victor Sgobbi Souza,
G. Solé,
L. Solera,
Suzara Souto Lopes,
S. Specht,
J. Statland,
A. Swenson,
C. Y. Tan,
S. Tizon,
N. V. Beek,
H. Kooten,
M. Wencel,
S. Wenninger,
F. Zagnoli
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6 |
2021 |
6 🐜
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🐢
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Maximum inspiratory pressure as a clinically meaningful trial endpoint for neuromuscular diseases: a comprehensive review of the literature
11 auth.
B. Schoser,
E. Fong,
T. Geberhiwot,
D. Hughes,
J. Kissel,
S. Madathil,
...
D. Orlikowski,
M. Polkey,
M. Roberts,
H. Tiddens,
P. Young
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6 |
2017 |
6 🐢
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Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascend
21 auth.
L. Pena,
R. Barohn,
B. Byrne,
C. Desnuelle,
O. Goker-Alpan,
S. Ladha,
P. Laforêt,
K. Mengel,
A. Pestronk,
J. Pouget,
...
B. Schoser,
V. Straub,
Jaya R. Trivedi,
P. van Damme,
J. Vissing,
P. Young,
K. Kacena,
R. Shafi,
B. Thurberg,
K. Culm-Merdek,
A. T. van der Ploeg
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6 |
2019 |
6 🐜
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Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
22 auth.
A. T. van der Ploeg,
P. Carlier,
R. Carlier,
J. Kissel,
B. Schoser,
S. Wenninger,
A. Pestronk,
R. Barohn,
M. Dimachkie,
O. Goker-Alpan,
...
T. Mozaffar,
L. Pena,
Z. Simmons,
V. Straub,
M. Guglieri,
P. Young,
M. Boentert,
P. Baudin,
S. Wens,
R. Shafi,
C. Bjartmar,
B. Thurberg
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5 |
2016 |
5 🐜
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🐢
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Sleep‐related symptoms and sleep‐disordered breathing in adult Pompe disease
7 auth.
M. Boentert,
N. Karabul,
S. Wenninger,
B. Stubbe-Dräger,
E. Mengel,
Benedikt Schoser,
...
P. Young
|
5 |
2015 |
5 🐢
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🐜
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A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A
19 auth.
S. Attarian,
P. Young,
T. Brannagan,
David John Adams,
P. van Damme,
F. Thomas,
Carlos Casanovas,
C. Tard,
M. Walter,
Y. Péréon,
...
D. Walk,
A. Stino,
M. de Visser,
C. Verhamme,
A. Amato,
Gregory Carter,
L. Magy,
J. Statland,
Kevin Felice
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5 |
2021 |
5 🐜
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Pulmonary function tests (maximum inspiratory pressure, maximum expiratory pressure, vital capacity, forced vital capacity) predict ventilator use in late-onset Pompe disease
E. Johnson,
M. Roberts,
T. Mozaffar,
P. Young,
A. Quartel,
K. Berger
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5 |
2016 |
5 🐬
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🐜
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The nature of respiratory muscle weakness in patients with late-onset Pompe disease
9 auth.
J. Spiesshoefer,
C. Henke,
H. Kabitz,
T. Brix,
D. Görlich,
S. Herkenrath,
...
W. Randerath,
P. Young,
M. Boentert
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4 |
2019 |
4 🐜
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