Replacing acid α-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers

Nina Raben(APT Therapeutics (United States)), Paul H. Plötz(National Institutes of Health), Kanneboyina Nagaraju(Children's National), Abigail L. Gilbert(National Institute of Arthritis and Musculoskeletal and Skin Diseases), Deborah de Jong(National Institutes of Health), Tokiko Fukuda(National Institutes of Health), Kunio Nagashima(Science Applications International Corporation (United States)), Beth L. Thurberg(Sanofi (United States)), Robert J. Mattaliano, John J. Hopwood(Women's and Children's Hospital), Peter J. Meikle(Baker Heart and Diabetes Institute)
Molecular Therapy
November 4, 2004
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