Effects of immune modulation therapy in the first Croatian infant diagnosed with Pompe disease: a 3-year follow-up study

Recombinant human acid α-glucosidase

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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

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A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

Priya S. Kishnani, Wuh‐Liang Hwu, Hanna Mandel et al. · The Journal of Pediatrics · 2006 · 578 citations

Pathogenesis, Natural History, Pediatrics +3

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Priya S. Kishnani, Paula Goldenberg, Stephanie DeArmey et al. · Molecular Genetics and Metabolism · 2009 · 372 citations

Vaccination, Clinical Immunology, Autoimmune Disease +10

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 et al. · Genetics in Medicine · 2001 · 346 citations · Full text

Translational Medicine, Biochemistry, Inherited Metabolic Disease +5