Mini-COMET study: Safety and efficacy data after avalglucosidase alfa dosing for ≥ 145 weeks in participants with infantile-onset Pompe disease (IOPD) who had demonstrated clinical decline or sub-optimal response while receiving alglucosidase alfa
David Kronn(New York Medical College), Yin‐Hsiu Chien(National Taiwan University Hospital), James Davison(Great Ormond Street Hospital for Children NHS Foundation Trust), Satoko Kumada(Tokyo Metropolitan Neurological Hospital), Swathi Tammireddy, Priya S. Kishnani(Duke Medical Center), Kristina An Haack(Sanofi (France)), Alexander Broomfield(St Mary's Hospital), Hirotaka Ohki(Tokyo Metropolitan Children's Medical Center), Susan Sparks(Children's National), Anaïs Brassier(Hôpital Necker-Enfants Malades), Tianyue Zhou, Atef Zaher(Sanofi (United States)), François Labarthe(Université de Tours), Sihoun Hahn(Mayo Clinic)
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