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Veröffentlichungsdatum
Anzahl der Publikationen: 3
2024
Schoser, Benedikt 
ORCID: https://orcid.org/0000-0002-2757-8131; Kishnani, Priya S.; Bratkovic, Drago; Byrne, Barry J.; Claeys, Kristl G.; Díaz-Manera, Jordi; Laforêt, Pascal; Roberts, Mark; Toscano, Antonio; Ploeg, Ans T. van der; Castelli, Jeff; Goldman, Mitchell; Holdbrook, Fred; Sitaraman Das, Sheela; Wasfi, Yasmine und Mozaffar, Tahseen
(28. Februar 2024):
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).
In: Journal of Neurology, Bd. 271, Nr. 5: S. 2810-2823
[PDF, 944kB]
ORCID: https://orcid.org/0000-0002-2757-8131; Kishnani, Priya S.; Bratkovic, Drago; Byrne, Barry J.; Claeys, Kristl G.; Díaz-Manera, Jordi; Laforêt, Pascal; Roberts, Mark; Toscano, Antonio; Ploeg, Ans T. van der; Castelli, Jeff; Goldman, Mitchell; Holdbrook, Fred; Sitaraman Das, Sheela; Wasfi, Yasmine und Mozaffar, Tahseen
(28. Februar 2024):
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).
In: Journal of Neurology, Bd. 271, Nr. 5: S. 2810-2823
[PDF, 944kB]
2021
Schoser, Benedikt; Roberts, Mark; Byrne, Barry J.; Sitaraman, Sheela; Jiang, Hai; Laforet, Pascal; Toscano, Antonio; Castelli, Jeff; Diaz-Manera, Jordi; Goldman, Mitchell; Ploeg, Ans T. van der; Bratkovic, Drago; Kuchipudi, Srilakshmi; Mozaffar, Tahseen und Kishnani, Priya S.
(2021):
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.
In: Lancet Neurology, Bd. 20, Nr. 12: S. 1027-1037
2019
Pena, Loren D. M.; Barohn, Richard J.; Byrne, Barry J.; Desnuelle, Claude; Goker-Alpan, Ozlem; Ladha, Shafeeq; Laforet, Pascal; Mengel, Karl Eugen; Pestronk, Alan; Pouget, Jean; Schoser, Benedikt; Straub, Volker; Trivedi, Jaya; Van Damme, Philip; Vissing, John; Young, Peter; Kacena, Katherine; Shafi, Raheel; Thurberg, Beth L.; Culm-Merdek, Kerry und van der Ploeg, Ans T.
(2019):
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
In: Neuromuscular Disorders, Bd. 29, Nr. 3: S. 167-186