Mini-COMET study: Safety, immunogenicity, and preliminary efficacy for repeat avalglucosidase alfa dosing in patients with infantile-onset Pompe disease (IOPD) who were previously treated with alglucosidase alfa and demonstrated clinical decline
David Kronn(New York Medical College), Judith Johnson(Sanofi (United States)), Satoko Kumada(Tokyo Metropolitan Neurological Hospital), Priya S. Kishnani(Duke Medical Center), Kristina An Haack(Sanofi (France)), Alexander Broomfield(St Mary's Hospital), C. Fleurinck(UCB Pharma (Belgium)), Hirotaka Ohki(Tokyo Metropolitan Children's Medical Center), Anaïs Brassier(Hôpital Necker-Enfants Malades), Shuang He(Central South University), Samia Pichard, Sihoun Hahn(Mayo Clinic), François Labarthe(Université de Tours), James Davison(Great Ormond Street Hospital for Children NHS Foundation Trust)
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