Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease

Zarate, 2008, Fabry’s disease., Lancet, 372, 1427, 10.1016/S0140-6736(08)61589-5

Eng, 2001, Safety and efficacy of recombinant human alpha-galactosidase A—replacement therapy in Fabry’s disease., N Engl J Med, 345, 9, 10.1056/NEJM200107053450102

Schiffmann, 2001, Enzyme replacement therapy in Fabry disease: a randomized controlled trial., JAMA, 285, 2743, 10.1001/jama.285.21.2743

Linthorst, 2004, Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta., Kidney Int, 66, 1589, 10.1111/j.1523-1755.2004.00924.x

Bénichou, 2009, A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease., Mol Genet Metab, 96, 4, 10.1016/j.ymgme.2008.10.004

Hollak, 2009, Immune response to enzyme replacement therapy in Fabry disease: impact on clinical outcome?, Mol Genet Metab, 96, 1, 10.1016/j.ymgme.2008.10.013

Rombach, 2012, Long-term effect of antibodies against infused alpha-galactosidase A in Fabry disease on plasma and urinary (lyso)Gb3 reduction and treatment outcome., PLoS ONE, 7, e47805, 10.1371/journal.pone.0047805

Lang, 2005, Recommendations for chamber quantification: a report from the American Society of Echocardiography’s Guidelines and Standards Committee and the Chamber Quantification Writing Group, developed in conjunction with the European Association of Echocardiography, a branch of the European Society of Cardiology., J Am Soc Echocardiogr, 18, 1440, 10.1016/j.echo.2005.10.005

Wilcox, 2012, Anti-α-galactosidase A antibody response to agalsidase beta treatment: data from the Fabry Registry., Mol Genet Metab, 105, 443, 10.1016/j.ymgme.2011.12.006

Tøndel, 2013, Agalsidase benefits renal histology in young patients with Fabry disease., J Am Soc Nephrol, 24, 137, 10.1681/ASN.2012030316

Warnock, 2014, Fabry disease: dose matters., J Am Soc Nephrol, 25, 653, 10.1681/ASN.2013121322

Weidemann, 2014, Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch., J Am Soc Nephrol, 25, 837, 10.1681/ASN.2013060585

Schellekens, 2008, The immunogenicity of therapeutic proteins and the Fabry antibody standardization initiative., Clin Ther, 30 Suppl B, S50, 10.1016/S0149-2918(08)80041-0

Brady, 1997, Management of neutralizing antibody to Ceredase in a patient with type 3 Gaucher disease., Pediatrics, 100, E11, 10.1542/peds.100.6.e11

Rosenberg, 1999, Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration., Blood, 93, 2081, 10.1182/blood.V93.6.2081.406k07_2081_2088

Markic, 2012, Immune modulation therapy in a CRIM-positive and IgG antibody-positive infant with Pompe disease treated with alglucosidase alpha: A case report., JIMD Rep, 2, 11, 10.1007/8904_2011_34

Brands, 2013, Mucopolysaccharidosis type VI phenotypes-genotypes and antibody response to galsulfase., Orphanet J Rare Dis, 8, 51, 10.1186/1750-1172-8-51

Douglas, 2011, ACCF/ASE/AHA/ASNC/HFSA/HRS/SCAI/SCCM/SCCT/SCMR 2011 Appropriate Use Criteria for Echocardiography. A Report of the American College of Cardiology Foundation Appropriate Use Criteria Task Force, American Society of Echocardiography, American Heart Association, American Society of Nuclear Cardiology, Heart Failure Society of America, Heart Rhythm Society, Society for Cardiovascular Angiography and Interventions, Society of Critical Care Medicine, Society of Cardiovascular Computed Tomography, and Society for Cardiovascular Magnetic Resonance Endorsed by the American College of Chest Physicians., J Am Coll Cardiol, 57, 1126, 10.1016/j.jacc.2010.11.002

Levey, 2009, A new equation to estimate glomerular filtration rate., Ann Intern Med, 150, 604, 10.7326/0003-4819-150-9-200905050-00006

Inker, 2012, Estimating glomerular filtration rate from serum creatinine and cystatin C., N Engl J Med, 367, 20, 10.1056/NEJMoa1114248

Whybra, 2004, The Mainz Severity Score Index: a new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy., Clin Genet, 65, 299, 10.1111/j.1399-0004.2004.00219.x

Giannini, 2010, A validated disease severity scoring system for Fabry disease., Mol Genet Metab, 99, 283, 10.1016/j.ymgme.2009.10.178

Desnick, 1973, Fabry’s disease: enzymatic diagnosis of hemizygotes and heterozygotes. Alpha-galactosidase activities in plasma, serum, urine, and leukocytes., J Lab Clin Med, 81, 157

Mayes, 1981, Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry’s disease., Clin Chim Acta, 112, 247, 10.1016/0009-8981(81)90384-3