2001•Genetics in MedicineOpen access

Recombinant human acid α-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial

Andrea Amalfitano, A. Resai Bengur, Richard P. Morse, Joseph M. Majure, Laura E. Case, Deborah L. Veerling, Joanne Mackey, Priya S. Kishnani, Wendy E. Smith, Alison McVie‐Wylie, Jennifer A. Sullivan, George Hoganson, John A. Phillips, G. Bradley Schaefer, Joel Charrow, Russell E. Ware, Edward H. Bossen, Yuan-Tsong Chen

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Key concepts: Recombinant DNA, Enzyme replacement therapy, Medicine, Enzyme, Glycogen storage disease, Glycogen storage disease type II, Clinical trial, Glycogen