Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
Top Cited Papers
- 31 January 2010
- journal article
- clinical trial
- Published by Elsevier BV in Molecular Genetics and Metabolism
- Vol. 99 (1), 26-33
- https://doi.org/10.1016/j.ymgme.2009.08.003
Abstract
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This publication has 44 references indexed in Scilit:
- Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease modelClinical and Experimental Immunology, 2008
- Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune ToleranceAmerican Journal of Human Genetics, 2007
- Differential muscular glycogen clearance after enzyme replacement therapy in a mouse model of Pompe diseaseMolecular Genetics and Metabolism, 2007
- Chinese hamster ovary cell-derived recombinant human acid α-glucosidase in infantile-onset Pompe diseaseThe Journal of Pediatrics, 2006
- Pompe disease diagnosis and management guidelineGenetics in Medicine, 2006
- Comparison of maltose and acarbose as inhibitors of maltase-glucoamylase activity in assaying acid α-glucosidase activity in dried blood spots for the diagnosis of infantile Pompe diseaseGenetics in Medicine, 2006
- Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)The Journal of Pediatrics, 2004
- Mutation analysis of the acid β-glucosidase gene in a patient with type 3 Gaucher disease and neutralizing antibody to algluceraseMutation research. Reviews in mutation research, 2001
- Factor IX Inhibitors and Anaphylaxis in Hemophilia BJournal of Pediatric Hematology/Oncology, 1997
- Nonparametric Estimation from Incomplete ObservationsJournal of the American Statistical Association, 1958