Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study
Si Houn Hahn(University of Washington), Priya S. Kishnani(Duke Medical Center), Raymond Wang(Children's Hospital of Orange County), Kristina An Haack(Sanofi (France)), Yang Zhao(Sanofi (United States)), Michael J. Gambello(Emory University), David W. Stockton(Baylor College of Medicine), Susan Sparks(Children's National), Raheel Shafi(Sanofi (United States)), Loren Peña(Cincinnati Children's Hospital Medical Center), James B. Gibson, Nancy D. Leslie(University of Cincinnati), David Kronn(New York Medical College), Pranoot Tanpaiboon(Children's National), Richard E. Hillman(University of Missouri Health System), Catherine Wilson(Murdoch Children's Research Institute)
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