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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 B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group. Schoser B, et al. J Neurol. 2024 Feb 28. doi: 10.1007/s00415-024-12236-0. Online ahead of print. J Neurol. 2024. PMID: 38418563
Benign tumors of the superficial lobe of the parotid gland.
Ştefănescu EH, Mogoantă CA, Căluianu EI, Predescu OI, Florou C, Chercotă V, Iovănescu G. Ştefănescu EH, et al. Rom J Morphol Embryol. 2022 Jul-Sep;63(3):563-567. doi: 10.47162/RJME.63.3.11. Rom J Morphol Embryol. 2022. PMID: 36588495 Free PMC article.
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.
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group. Schoser B, et al. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Lancet Neurol. 2021. PMID: 34800400 Clinical Trial.
77 results