Antibody-mediated enzyme replacement therapy targeting both lysosomal and cytoplasmic glycogen in Pompe disease

[1] van der Ploeg AT(2008)Pompe’s disease Lancet 372 1342-1353

[2] Reuser AJ(2006)A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease J Pediatr 148 671-676

[3] Kishnani PS(2007)Late onset Pompe disease: clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients Neuromuscul Disord 17 698-706

[4] Hwu WL(2012)The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management Am J Med Genet C Semin Med Genet 160 1-7

[5] Mandel H(2012)Enzyme replacement therapy for Pompe disease Curr Neurol Neurosci Rep 12 70-75

[6] Nicolino M(2012)Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations Am J Med Genet C Semin Med Genet 160 69-79

[7] Yong F(1996)High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease Proc Natl Acad Sci U S A 93 65-70

[8] Corzo D(1968)Type II glycogenosis. Biochemical and electron microscopic study Am J Med 44 289-300

[9] Muller-Felber W(2006)Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease Lab Invest 86 1208-1220

[10] Horvath R(2008)Pathology of skeletal muscle cells in adult-onset glycogenosis type II (Pompe disease): ultrastructural study Folia Neuropathol 46 123-133

[1] van der Ploeg AT(2008)Pompe’s disease Lancet 372 1342-1353

[2] Reuser AJ(2006)A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease J Pediatr 148 671-676

[3] Kishnani PS(2007)Late onset Pompe disease: clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients Neuromuscul Disord 17 698-706

[4] Hwu WL(2012)The new era of Pompe disease: advances in the detection, understanding of the phenotypic spectrum, pathophysiology, and management Am J Med Genet C Semin Med Genet 160 1-7

[5] Mandel H(2012)Enzyme replacement therapy for Pompe disease Curr Neurol Neurosci Rep 12 70-75

[6] Nicolino M(2012)Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations Am J Med Genet C Semin Med Genet 160 69-79

[7] Yong F(1996)High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease Proc Natl Acad Sci U S A 93 65-70

[8] Corzo D(1968)Type II glycogenosis. Biochemical and electron microscopic study Am J Med 44 289-300

[9] Muller-Felber W(2006)Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease Lab Invest 86 1208-1220

[10] Horvath R(2008)Pathology of skeletal muscle cells in adult-onset glycogenosis type II (Pompe disease): ultrastructural study Folia Neuropathol 46 123-133