Efficacy and safety of avalglucosidase alfa in Japanese patients with late-onset and infantile-onset Pompe diseases: A case series from clinical trials

Efficacy and safety of avalglucosidase alfa in Japanese patients with late-onset and infantile-onset Pompe diseases: A case series from clinical trials

Background: The efficacy and safety of avalglucosidase alfa for Pompe disease (PD) have been demonstrated in a global Phase 3 trial (COMET) in patients with late-onset PD (LOPD) and a global Phase 2 trial (Mini-COMET) in patients with infantile-onset PD (IOPD). This case series examines the individu...

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Main Authors: Madoka Mori-Yoshimura, Hirotaka Ohki, Hideaki Mashimo, Kenji Inoue, Satoko Kumada, Takashi Kiyono, Akihiro Arimori, Mitsunobu Ikeda, Hirofumi Komaki
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Avalglucosidase alfa
Case series
Enzyme replacement therapy
Japanese
Infantile-onset Pompe disease
Late-onset Pompe disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426924001162
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