{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,26]],"date-time":"2026-10-26T10:47:08Z","timestamp":1793011628030,"version":"4.5.0"},"reference-count":122,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"9","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JASN"],"published-print":{"date-parts":[[2018,9]]},"DOI":"10.1681\/asn.2018030329","type":"journal-article","created":{"date-parts":[[2018,8,9]],"date-time":"2018-08-09T12:55:42Z","timestamp":1533819342000},"page":"2265-2278","source":"Crossref","is-referenced-by-count":91,"title":["Effects of Enzyme Replacement Therapy and Antidrug Antibodies in Patients with Fabry Disease"],"prefix":"10.1681","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8409-2886","authenticated-orcid":false,"given":"Malte","family":"Lenders","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva","family":"Brand","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","published-online":{"date-parts":[[2018,8,9]]},"reference":[{"key":"B1-20230829","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","article-title":"Prevalence of lysosomal storage disorders","volume":"281","author":"Meikle","year":"1999","journal-title":"JAMA"},{"key":"B2-20230829","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/B978-0-444-62702-5.00017-2","article-title":"Fabry disease","volume":"132","author":"Schiffmann","year":"2015","journal-title":"Handb Clin Neurol"},{"key":"B3-20230829","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.1016\/S0140-6736(08)61589-5","article-title":"Fabry\u2019s disease","volume":"372","author":"Zarate","year":"2008","journal-title":"Lancet"},{"key":"B4-20230829","doi-asserted-by":"crossref","first-page":"1906","DOI":"10.1016\/j.cca.2010.07.038","article-title":"How well does urinary lyso-Gb3 function as a biomarker in Fabry disease?","volume":"411","author":"Auray-Blais","year":"2010","journal-title":"Clin Chim Acta"},{"key":"B5-20230829","doi-asserted-by":"crossref","first-page":"8","DOI":"10.1161\/CIRCGENETICS.113.000249","article-title":"Gene mutations versus clinically relevant phenotypes: Lyso-Gb3 defines Fabry disease","volume":"7","author":"Niemann","year":"2014","journal-title":"Circ Cardiovasc Genet"},{"key":"B6-20230829","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/j.cca.2014.08.002","article-title":"Urinary biomarker investigation in children with Fabry disease using tandem mass spectrometry","volume":"438","author":"Auray-Blais","year":"2015","journal-title":"Clin Chim Acta"},{"key":"B7-20230829","doi-asserted-by":"crossref","first-page":"13382","DOI":"10.1021\/acs.analchem.7b03609","article-title":"Separation and analysis of lactosylceramide, galabiosylceramide, and globotriaosylceramide by LC-MS\/MS in urine of Fabry disease patients","volume":"89","author":"Boutin","year":"2017","journal-title":"Anal Chem"},{"key":"B8-20230829","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1016\/j.ymgme.2016.10.006","article-title":"Plasma LysoGb3: A useful biomarker for the diagnosis and treatment of Fabry disease heterozygotes","volume":"120","author":"Nowak","year":"2017","journal-title":"Mol Genet Metab"},{"key":"B9-20230829","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1016\/j.cca.2017.01.018","article-title":"Biomarkers associated with clinical manifestations in Fabry disease patients with a late-onset cardiac variant mutation","volume":"466","author":"Auray-Blais","year":"2017","journal-title":"Clin Chim Acta"},{"key":"B10-20230829","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/j.ymgme.2017.05.001","article-title":"Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease","volume":"121","author":"Arends","year":"2017","journal-title":"Mol Genet Metab"},{"key":"B11-20230829","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1136\/jmg.38.11.750","article-title":"Anderson-Fabry disease: Clinical manifestations and impact of disease in a cohort of 98 hemizygous males","volume":"38","author":"MacDermot","year":"2001","journal-title":"J Med Genet"},{"key":"B12-20230829","doi-asserted-by":"crossref","first-page":"769","DOI":"10.1136\/jmg.38.11.769","article-title":"Anderson-Fabry disease: Clinical manifestations and impact of disease in a cohort of 60 obligate carrier females","volume":"38","author":"MacDermot","year":"2001","journal-title":"J Med Genet"},{"key":"B13-20230829","doi-asserted-by":"crossref","first-page":"790","DOI":"10.1097\/GIM.0b013e3181bb05bb","article-title":"Life expectancy and cause of death in males and females with Fabry disease: Findings from the Fabry Registry","volume":"11","author":"Waldek","year":"2009","journal-title":"Genet Med"},{"key":"B14-20230829","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1186\/s13023-016-0473-4","article-title":"Multicenter Female Fabry Study (MFFS) - clinical survey on current treatment of females with Fabry disease","volume":"11","author":"Lenders","year":"2016","journal-title":"Orphanet J Rare Dis"},{"key":"B15-20230829","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1111\/cge.12613","article-title":"X-chromosome inactivation in female patients with Fabry disease","volume":"89","author":"Echevarria","year":"2016","journal-title":"Clin Genet"},{"key":"B17-20230829","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1016\/j.ijcard.2014.09.001","article-title":"Uncertain diagnosis of Fabry disease: Consensus recommendation on diagnosis in adults with left ventricular hypertrophy and genetic variants of unknown significance","volume":"177","author":"Smid","year":"2014","journal-title":"Int J Cardiol"},{"key":"B18-20230829","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1186\/s13023-015-0253-6","article-title":"Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: The European Fabry Working Group consensus document","volume":"10","author":"Biegstraaten","year":"2015","journal-title":"Orphanet J Rare Dis"},{"key":"B19-20230829","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1056\/NEJM200107053450102","article-title":"Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry\u2019s disease","volume":"345","author":"Eng","year":"2001","journal-title":"N Engl J Med"},{"key":"B20-20230829","doi-asserted-by":"crossref","first-page":"2743","DOI":"10.1001\/jama.285.21.2743","article-title":"Enzyme replacement therapy in Fabry disease: A randomized controlled trial","volume":"285","author":"Schiffmann","year":"2001","journal-title":"JAMA"},{"key":"B21-20230829","doi-asserted-by":"crossref","first-page":"545","DOI":"10.1056\/NEJMoa1510198","article-title":"Treatment of Fabry\u2019s disease with the pharmacologic chaperone migalastat","volume":"375","author":"Germain","year":"2016","journal-title":"N Engl J Med"},{"key":"B22-20230829","doi-asserted-by":"crossref","first-page":"1589","DOI":"10.1111\/j.1523-1755.2004.00924.x","article-title":"Enzyme therapy for Fabry disease: Neutralizing antibodies toward agalsidase alpha and beta","volume":"66","author":"Linthorst","year":"2004","journal-title":"Kidney Int"},{"key":"B23-20230829","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1016\/j.ymgme.2008.03.003","article-title":"Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3","volume":"94","author":"Vedder","year":"2008","journal-title":"Mol Genet Metab"},{"key":"B24-20230829","doi-asserted-by":"crossref","first-page":"e47805","DOI":"10.1371\/journal.pone.0047805","article-title":"Long-term effect of antibodies against infused alpha-galactosidase A in Fabry disease on plasma and urinary (lyso)Gb3 reduction and treatment outcome","volume":"7","author":"Rombach","year":"2012","journal-title":"PLoS One"},{"key":"B25-20230829","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1681\/ASN.2014121226","article-title":"Serum-mediated inhibition of enzyme replacement therapy in Fabry disease","volume":"27","author":"Lenders","year":"2016","journal-title":"J Am Soc Nephrol"},{"key":"B26-20230829","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/j.ymgme.2014.08.002","article-title":"Characterization of a chemically modified plant cell culture expressed human \u03b1-Galactosidase-A enzyme for treatment of Fabry disease","volume":"114","author":"Kizhner","year":"2015","journal-title":"Mol Genet Metab"},{"key":"B27-20230829","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1007\/s10545-015-9886-9","article-title":"Mannose receptor-mediated delivery of moss-made \u03b1-galactosidase A efficiently corrects enzyme deficiency in Fabry mice","volume":"39","author":"Shen","year":"2016","journal-title":"J Inherit Metab Dis"},{"key":"B28-20230829","first-page":"77","article-title":"Enzyme replacement therapy and renal function in 201 patients with Fabry disease","volume":"66","author":"Schwarting","year":"2006","journal-title":"Clin Nephrol"},{"key":"B29-20230829","doi-asserted-by":"crossref","first-page":"1576","DOI":"10.1681\/ASN.2006111263","article-title":"Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing","volume":"18","author":"Schiffmann","year":"2007","journal-title":"J Am Soc Nephrol"},{"key":"B30-20230829","doi-asserted-by":"crossref","first-page":"77","DOI":"10.7326\/0003-4819-146-2-200701160-00148","article-title":"Agalsidase-beta therapy for advanced Fabry disease: A randomized trial","volume":"146","author":"Banikazemi","year":"2007","journal-title":"Ann Intern Med"},{"key":"B31-20230829","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1016\/j.ymgmr.2015.02.002","article-title":"Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis","volume":"3","author":"Beck","year":"2015","journal-title":"Mol Genet Metab Rep"},{"key":"B32-20230829","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1186\/s13023-015-0338-2","article-title":"Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: Cardiac outcomes after 10 years\u2019 treatment","volume":"10","author":"Kampmann","year":"2015","journal-title":"Orphanet J Rare Dis"},{"key":"B33-20230829","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1136\/jmedgenet-2014-102797","article-title":"Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease","volume":"52","author":"Germain","year":"2015","journal-title":"J Med Genet"},{"key":"B34-20230829","doi-asserted-by":"crossref","first-page":"1129","DOI":"10.1007\/s10545-015-9845-5","article-title":"A prospective 10-year study of individualized, intensified enzyme replacement therapy in advanced Fabry disease","volume":"38","author":"Schiffmann","year":"2015","journal-title":"J Inherit Metab Dis"},{"key":"B35-20230829","doi-asserted-by":"crossref","first-page":"952","DOI":"10.1681\/ASN.2015030337","article-title":"Patients with Fabry disease after enzyme replacement therapy dose reduction and switch -2-year follow-up","volume":"27","author":"Lenders","year":"2016","journal-title":"J Am Soc Nephrol"},{"key":"B36-20230829","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1136\/jmedgenet-2017-104863","article-title":"Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: An international cohort study","volume":"55","author":"Arends","year":"2018","journal-title":"J Med Genet"},{"key":"B37-20230829","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1097\/00005792-200203000-00003","article-title":"Natural history of Fabry renal disease: Influence of alpha-galactosidase A activity and genetic mutations on clinical course","volume":"81","author":"Branton","year":"2002","journal-title":"Medicine (Baltimore)"},{"key":"B38-20230829","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1681\/ASN.2012030316","article-title":"Agalsidase benefits renal histology in young patients with Fabry disease","volume":"24","author":"T\u00f8ndel","year":"2013","journal-title":"J Am Soc Nephrol"},{"key":"B39-20230829","doi-asserted-by":"crossref","first-page":"e0152812","DOI":"10.1371\/journal.pone.0152812","article-title":"One year of enzyme replacement therapy reduces globotriaosylceramide inclusions in podocytes in male adult patients with Fabry disease","volume":"11","author":"Najafian","year":"2016","journal-title":"PLoS One"},{"key":"B40-20230829","first-page":"807","article-title":"Reaccumulation of globotriaosylceramide in podocytes after agalsidase dose reduction in young Fabry patients","volume":"32","author":"Skrunes","year":"2017","journal-title":"Nephrol Dial Transplant"},{"key":"B41-20230829","doi-asserted-by":"crossref","first-page":"1470","DOI":"10.2215\/CJN.01820217","article-title":"Long-term dose-dependent agalsidase effects on kidney histology in Fabry disease","volume":"12","author":"Skrunes","year":"2017","journal-title":"Clin J Am Soc Nephrol"},{"key":"B42-20230829","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1111\/j.1365-2362.2004.01424.x","article-title":"Fabry disease: Overall effects of agalsidase alfa treatment","volume":"34","author":"Beck","year":"2004","journal-title":"Eur J Clin Invest"},{"key":"B43-20230829","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1093\/ndt\/gfi152","article-title":"Long-term therapy with agalsidase alfa for Fabry disease: Safety and effects on renal function in a home infusion setting","volume":"21","author":"Schiffmann","year":"2006","journal-title":"Nephrol Dial Transplant"},{"key":"B44-20230829","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1159\/000168482","article-title":"Agalsidase alfa slows the decline in renal function in patients with Fabry disease","volume":"29","author":"Feriozzi","year":"2009","journal-title":"Am J Nephrol"},{"key":"B45-20230829","doi-asserted-by":"crossref","first-page":"1132","DOI":"10.1681\/ASN.2008080870","article-title":"Agalsidase alfa and kidney dysfunction in Fabry disease","volume":"20","author":"West","year":"2009","journal-title":"J Am Soc Nephrol"},{"key":"B46-20230829","doi-asserted-by":"crossref","first-page":"60","DOI":"10.2215\/CJN.03130411","article-title":"The effectiveness of long-term agalsidase alfa therapy in the treatment of Fabry nephropathy","volume":"7","author":"Feriozzi","year":"2012","journal-title":"Clin J Am Soc Nephrol"},{"key":"B47-20230829","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1186\/s13023-014-0169-6","article-title":"Agalsidase alfa in pediatric patients with Fabry disease: A 6.5-year open-label follow-up study","volume":"9","author":"Schiffmann","year":"2014","journal-title":"Orphanet J Rare Dis"},{"key":"B48-20230829","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1086\/422366","article-title":"Long-term safety and efficacy of enzyme replacement therapy for Fabry disease","volume":"75","author":"Wilcox","year":"2004","journal-title":"Am J Hum Genet"},{"key":"B49-20230829","doi-asserted-by":"crossref","first-page":"1216","DOI":"10.1038\/sj.ki.5000208","article-title":"Clinical benefit of enzyme replacement therapy in Fabry disease","volume":"69","author":"Breunig","year":"2006","journal-title":"Kidney Int"},{"key":"B50-20230829","doi-asserted-by":"crossref","first-page":"1547","DOI":"10.1681\/ASN.2006080816","article-title":"Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease","volume":"18","author":"Germain","year":"2007","journal-title":"J Am Soc Nephrol"},{"key":"B51-20230829","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1093\/ndt\/gfr420","article-title":"Renal outcomes of agalsidase beta treatment for Fabry disease: Role of proteinuria and timing of treatment initiation","volume":"27","author":"Warnock","year":"2012","journal-title":"Nephrol Dial Transplant"},{"key":"B52-20230829","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1681\/ASN.2013060585","article-title":"Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch","volume":"25","author":"Weidemann","year":"2014","journal-title":"J Am Soc Nephrol"},{"key":"B53-20230829","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/j.beem.2014.10.003","article-title":"Fabry disease and the heart","volume":"29","author":"Seydelmann","year":"2015","journal-title":"Best Pract Res Clin Endocrinol Metab"},{"key":"B54-20230829","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1023\/B:BOLI.0000005658.14563.77","article-title":"Enzyme replacement therapy in heterozygous females with Fabry disease: Results of a phase IIIB study","volume":"26","author":"Baehner","year":"2003","journal-title":"J Inherit Metab Dis"},{"key":"B55-20230829","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1136\/hrt.2006.104026","article-title":"Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: A randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa","volume":"94","author":"Hughes","year":"2008","journal-title":"Heart"},{"key":"B56-20230829","doi-asserted-by":"crossref","first-page":"1986","DOI":"10.1016\/S0140-6736(09)61493-8","article-title":"Enzyme replacement therapy with agalsidase alfa in patients with Fabry\u2019s disease: An analysis of registry data","volume":"374","author":"Mehta","year":"2009","journal-title":"Lancet"},{"key":"B57-20230829","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1097\/GIM.0b013e3181a23bec","article-title":"A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease","volume":"11","author":"Whybra","year":"2009","journal-title":"Genet Med"},{"key":"B58-20230829","doi-asserted-by":"crossref","first-page":"1299","DOI":"10.1161\/01.CIR.0000091253.71282.04","article-title":"Improvement of cardiac function during enzyme replacement therapy in patients with Fabry disease: A prospective strain rate imaging study","volume":"108","author":"Weidemann","year":"2003","journal-title":"Circulation"},{"key":"B59-20230829","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1111\/j.1399-0004.2004.00284.x","article-title":"Enzyme replacement therapy with agalsidase beta improves cardiac involvement in Fabry\u2019s disease","volume":"66","author":"Spinelli","year":"2004","journal-title":"Clin Genet"},{"key":"B60-20230829","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1136\/hrt.2008.162800","article-title":"Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: A prospective long-term cardiac magnetic resonance imaging study","volume":"95","author":"Imbriaco","year":"2009","journal-title":"Heart"},{"key":"B61-20230829","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1161\/CIRCULATIONAHA.108.794529","article-title":"Long-term effects of enzyme replacement therapy on fabry cardiomyopathy: Evidence for a better outcome with early treatment","volume":"119","author":"Weidemann","year":"2009","journal-title":"Circulation"},{"key":"B62-20230829","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1093\/eurjhf\/hfq211","article-title":"Cardiac energy metabolism is disturbed in Fabry disease and improves with enzyme replacement therapy using recombinant human galactosidase A","volume":"13","author":"Machann","year":"2011","journal-title":"Eur J Heart Fail"},{"key":"B63-20230829","doi-asserted-by":"crossref","first-page":"1506","DOI":"10.1161\/hc3801.096352","article-title":"Regional cerebral hyperperfusion and nitric oxide pathway dysregulation in Fabry disease: Reversal by enzyme replacement therapy","volume":"104","author":"Moore","year":"2001","journal-title":"Circulation"},{"key":"B64-20230829","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1186\/1471-2377-2-4","article-title":"Enzyme replacement reverses abnormal cerebrovascular responses in Fabry disease","volume":"2","author":"Moore","year":"2002","journal-title":"BMC Neurol"},{"key":"B65-20230829","doi-asserted-by":"crossref","first-page":"1009","DOI":"10.1212\/WNL.0000000000001333","article-title":"Thromboembolic events in Fabry disease and the impact of factor V Leiden","volume":"84","author":"Lenders","year":"2015","journal-title":"Neurology"},{"key":"B66-20230829","doi-asserted-by":"crossref","first-page":"e0173358","DOI":"10.1371\/journal.pone.0173358","article-title":"Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies","volume":"12","author":"El Dib","year":"2017","journal-title":"PLoS One"},{"key":"B67-20230829","doi-asserted-by":"crossref","first-page":"525","DOI":"10.1161\/hs0202.102601","article-title":"Elevated cerebral blood flow velocities in Fabry disease with reversal after enzyme replacement","volume":"33","author":"Moore","year":"2002","journal-title":"Stroke"},{"key":"B68-20230829","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1159\/000369293","article-title":"Enzyme replacement therapy stabilized white matter lesion progression in Fabry disease","volume":"38","author":"Fellgiebel","year":"2014","journal-title":"Cerebrovasc Dis"},{"key":"B69-20230829","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1136\/jmg.2004.025791","article-title":"Effects of enzyme replacement therapy on pain and health related quality of life in patients with Fabry disease: Data from FOS (Fabry Outcome Survey)","volume":"42","author":"Hoffmann","year":"2005","journal-title":"J Med Genet"},{"key":"B70-20230829","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1097\/AJP.0b013e318074c986","article-title":"Nature and prevalence of pain in Fabry disease and its response to enzyme replacement therapy--a retrospective analysis from the Fabry Outcome Survey","volume":"23","author":"Hoffmann","year":"2007","journal-title":"Clin J Pain"},{"key":"B71-20230829","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1212\/01.WNL.0000118207.84514.40","article-title":"Enzyme replacement therapy improves function of C-, Adelta-, and Abeta-nerve fibers in Fabry neuropathy","volume":"62","author":"Hilz","year":"2004","journal-title":"Neurology"},{"key":"B72-20230829","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1023\/B:BOLI.0000037342.59612.69","article-title":"Relief of gastrointestinal symptoms under enzyme replacement therapy [corrected] in patients with Fabry disease","volume":"27","author":"Dehout","year":"2004","journal-title":"J Inherit Metab Dis"},{"key":"B73-20230829","doi-asserted-by":"crossref","first-page":"1447","DOI":"10.1016\/j.cgh.2007.08.012","article-title":"Gastrointestinal symptoms in 342 patients with Fabry disease: Prevalence and response to enzyme replacement therapy","volume":"5","author":"Hoffmann","year":"2007","journal-title":"Clin Gastroenterol Hepatol"},{"key":"B74-20230829","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.ymgme.2005.04.009","article-title":"Gastrointestinal manifestations of Fabry disease: Clinical response to enzyme replacement therapy","volume":"85","author":"Banikazemi","year":"2005","journal-title":"Mol Genet Metab"},{"key":"B75-20230829","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1007\/8904_2017_28","article-title":"Improvement of Fabry disease-related gastrointestinal symptoms in a significant proportion of female patients treated with agalsidase beta: Data from the Fabry Registry","volume":"38","author":"Wilcox","year":"2018","journal-title":"JIMD Rep"},{"key":"B76-20230829","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1111\/j.1399-0004.2004.00219.x","article-title":"The Mainz Severity Score Index: A new instrument for quantifying the Anderson-Fabry disease phenotype, and the response of patients to enzyme replacement therapy","volume":"65","author":"Whybra","year":"2004","journal-title":"Clin Genet"},{"key":"B77-20230829","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.ymgme.2009.10.178","article-title":"A validated disease severity scoring system for Fabry disease","volume":"99","author":"Giannini","year":"2010","journal-title":"Mol Genet Metab"},{"key":"B78-20230829","doi-asserted-by":"crossref","first-page":"739","DOI":"10.1093\/ckj\/sfw082","article-title":"FAbry STabilization indEX (FASTEX): An innovative tool for the assessment of clinical stabilization in Fabry disease","volume":"9","author":"Mignani","year":"2016","journal-title":"Clin Kidney J"},{"key":"B79-20230829","doi-asserted-by":"crossref","first-page":"860","DOI":"10.1136\/jmedgenet-2015-103471","article-title":"Antiproteinuric therapy and Fabry nephropathy: Factors associated with preserved kidney function during agalsidase-beta therapy","volume":"52","author":"Warnock","year":"2015","journal-title":"J Med Genet"},{"key":"B80-20230829","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1007\/s10545-014-9677-8","article-title":"Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: A systematic review and meta-analysis: Effectiveness of ERT in different disease stages","volume":"37","author":"Rombach","year":"2014","journal-title":"J Inherit Metab Dis"},{"key":"B82-20230829","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1136\/jmedgenet-2016-104178","article-title":"Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study","volume":"54","author":"Hughes","year":"2017","journal-title":"J Med Genet"},{"key":"B83-20230829","doi-asserted-by":"crossref","first-page":"389","DOI":"10.2119\/molmed.2015.00088","article-title":"Efficacy of enzyme and substrate reduction therapy with a novel antagonist of glucosylceramide synthase for Fabry disease","volume":"21","author":"Ashe","year":"2015","journal-title":"Mol Med"},{"key":"B84-20230829","doi-asserted-by":"crossref","first-page":"703","DOI":"10.1002\/cpt.790","article-title":"Lucerastat, an iminosugar for substrate reduction therapy: Tolerability, pharmacodynamics, and pharmacokinetics in patients with Fabry disease on enzyme replacement","volume":"103","author":"Gu\u00e9rard","year":"2018","journal-title":"Clin Pharmacol Ther"},{"key":"B85-20230829","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1016\/j.omtm.2017.05.003","article-title":"Lentivector iterations and pre-clinical scale-up\/toxicity testing: Targeting mobilized CD34+ cells for correction of Fabry disease","volume":"5","author":"Huang","year":"2017","journal-title":"Mol Ther Methods Clin Dev"},{"key":"B86-20230829","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1056\/NEJM199302183280701","article-title":"Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors","volume":"328","author":"Lusher","year":"1993","journal-title":"N Engl J Med"},{"key":"B87-20230829","doi-asserted-by":"crossref","first-page":"901","DOI":"10.1038\/nbt.1484","article-title":"Neutralizing antibodies to therapeutic enzymes: Considerations for testing, prevention and treatment","volume":"26","author":"Wang","year":"2008","journal-title":"Nat Biotechnol"},{"key":"B88-20230829","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1016\/j.ymgme.2010.08.009","article-title":"High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa","volume":"101","author":"de Vries","year":"2010","journal-title":"Mol Genet Metab"},{"key":"B89-20230829","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/j.ymgme.2011.12.006","article-title":"Anti-\u03b1-galactosidase A antibody response to agalsidase beta treatment: Data from the Fabry Registry","volume":"105","author":"Wilcox","year":"2012","journal-title":"Mol Genet Metab"},{"key":"B90-20230829","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1016\/j.ymgme.2012.12.005","article-title":"A revised home treatment algorithm for Fabry disease: Influence of antibody formation","volume":"108","author":"Smid","year":"2013","journal-title":"Mol Genet Metab"},{"key":"B91-20230829","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1007\/8904_2011_106","article-title":"Severe infusion reactions to fabry enzyme replacement therapy: Rechallenge after tracheostomy","volume":"5","author":"Nicholls","year":"2012","journal-title":"JIMD Rep"},{"key":"B92-20230829","first-page":"367","article-title":"Successful desensitization with agalsidase alfa in 2 brothers with Fabry disease","volume":"23","author":"Aydin","year":"2013","journal-title":"J Investig Allergol Clin Immunol"},{"issue":"Suppl 3","key":"B95-20230829","doi-asserted-by":"crossref","first-page":"S249","DOI":"10.1007\/s10545-010-9136-0","article-title":"Enzyme replacement therapy in a patient with Fabry disease and the development of IgE antibodies against agalsidase beta but not agalsidase alpha","volume":"33","author":"Tanaka","year":"2010","journal-title":"J Inherit Metab Dis"},{"key":"B96-20230829","doi-asserted-by":"crossref","first-page":"563","DOI":"10.1016\/j.jpeds.2007.09.007","article-title":"Safety and efficacy of enzyme replacement therapy with agalsidase beta: An international, open-label study in pediatric patients with Fabry disease","volume":"152","author":"Wraith","year":"2008","journal-title":"J Pediatr"},{"key":"B97-20230829","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1097\/GIM.0b013e318170f868","article-title":"Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme","volume":"10","author":"Bodensteiner","year":"2008","journal-title":"Genet Med"},{"key":"B98-20230829","doi-asserted-by":"crossref","first-page":"15053","DOI":"10.1038\/mtm.2015.53","article-title":"Targeted approaches to induce immune tolerance for Pompe disease therapy","volume":"3","author":"Doerfler","year":"2016","journal-title":"Mol Ther Methods Clin Dev"},{"key":"B99-20230829","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1046\/j.1365-2222.2003.01765.x","article-title":"Induction of \u2018blocking\u2019 IgG antibodies during immunotherapy","volume":"33","author":"Wachholz","year":"2003","journal-title":"Clin Exp Allergy"},{"key":"B100-20230829","doi-asserted-by":"crossref","first-page":"915","DOI":"10.1016\/S0091-6749(03)02022-0","article-title":"Inhibition of allergen-IgE binding to B cells by IgG antibodies after grass pollen immunotherapy","volume":"112","author":"Wachholz","year":"2003","journal-title":"J Allergy Clin Immunol"},{"key":"B101-20230829","doi-asserted-by":"crossref","first-page":"833","DOI":"10.1172\/JCI25575","article-title":"IgG-blocking antibodies inhibit IgE-mediated anaphylaxis in vivo through both antigen interception and Fc gamma RIIb cross-linking","volume":"116","author":"Strait","year":"2006","journal-title":"J Clin Invest"},{"key":"B102-20230829","doi-asserted-by":"crossref","first-page":"2289","DOI":"10.1016\/j.jaci.2017.12.1001","article-title":"Characterization of drug-neutralizing antibodies in patients with Fabry disease during infusion","volume":"141","author":"Lenders","year":"2018","journal-title":"J Allergy Clin Immunol"},{"key":"B103-20230829","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1097\/GIM.0b013e3181f13b75","article-title":"Effects of enzyme replacement therapy in Fabry disease--a comprehensive review of the medical literature","volume":"12","author":"Lidove","year":"2010","journal-title":"Genet Med"},{"key":"B104-20230829","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/8904_2014_371","article-title":"Innate and adaptive immune response in Fabry disease","volume":"22","author":"Mauhin","year":"2015","journal-title":"JIMD Rep"},{"key":"B105-20230829","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1007\/BF03261891","article-title":"Agalsidase alfa: A review of its use in the management of Fabry disease","volume":"26","author":"Keating","year":"2012","journal-title":"BioDrugs"},{"key":"B106-20230829","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.ymgme.2008.10.004","article-title":"A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease","volume":"96","author":"B\u00e9nichou","year":"2009","journal-title":"Mol Genet Metab"},{"key":"B107-20230829","doi-asserted-by":"crossref","first-page":"e25065","DOI":"10.1371\/journal.pone.0025065","article-title":"Receptor-mediated endocytosis of \u03b1-galactosidase A in human podocytes in Fabry disease","volume":"6","author":"Prabakaran","year":"2011","journal-title":"PLoS One"},{"key":"B108-20230829","doi-asserted-by":"crossref","first-page":"496","DOI":"10.1111\/j.1365-2249.2004.02567.x","article-title":"Methotrexate reduces antibody responses to recombinant human alpha-galactosidase A therapy in a mouse model of Fabry disease","volume":"137","author":"Garman","year":"2004","journal-title":"Clin Exp Immunol"},{"key":"B109-20230829","doi-asserted-by":"crossref","first-page":"56","DOI":"10.1016\/j.clim.2017.01.014","article-title":"Anti-BlyS antibody reduces the immune reaction against enzyme and enhances the efficacy of enzyme replacement therapy in Fabry disease model mice","volume":"178","author":"Sato","year":"2017","journal-title":"Clin Immunol"},{"key":"B110-20230829","doi-asserted-by":"crossref","first-page":"3947","DOI":"10.4049\/jimmunol.1303326","article-title":"Transient low-dose methotrexate generates B regulatory cells that mediate antigen-specific tolerance to alglucosidase alfa","volume":"193","author":"Joly","year":"2014","journal-title":"J Immunol"},{"key":"B111-20230829","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1007\/8904_2011_34","article-title":"Immune modulation therapy in a CRIM-positive and IgG antibody-positive infant with Pompe disease treated with alglucosidase alpha: A case report","volume":"2","author":"Markic","year":"2012","journal-title":"JIMD Rep"},{"key":"B112-20230829","doi-asserted-by":"crossref","first-page":"847","DOI":"10.1016\/j.jpeds.2013.03.002","article-title":"B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease","volume":"163","author":"Elder","year":"2013","journal-title":"J Pediatr"},{"key":"B113-20230829","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1038\/gim.2012.110","article-title":"Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: Lessons learned from Pompe disease","volume":"15","author":"Banugaria","year":"2013","journal-title":"Genet Med"},{"key":"B114-20230829","doi-asserted-by":"crossref","first-page":"e94328","DOI":"10.1172\/jci.insight.94328","article-title":"Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease","volume":"2","author":"Kazi","year":"2017","journal-title":"JCI Insight"},{"key":"B115-20230829","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1111\/joim.12647","article-title":"Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients with Fabry disease","volume":"282","author":"Lenders","year":"2017","journal-title":"J Intern Med"},{"key":"B116-20230829","doi-asserted-by":"crossref","first-page":"E11","DOI":"10.1542\/peds.100.6.e11","article-title":"Management of neutralizing antibody to Ceredase in a patient with type 3 Gaucher disease","volume":"100","author":"Brady","year":"1997","journal-title":"Pediatrics"},{"key":"B117-20230829","doi-asserted-by":"crossref","first-page":"e67052","DOI":"10.1371\/journal.pone.0067052","article-title":"Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: A step towards improving the efficacy of ERT","volume":"8","author":"Banugaria","year":"2013","journal-title":"PLoS One"},{"key":"B118-20230829","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1007\/s00508-013-0475-3","article-title":"Effects of immune modulation therapy in the first Croatian infant diagnosed with Pompe disease: A 3-year follow-up study","volume":"126","author":"Markic","year":"2014","journal-title":"Wien Klin Wochenschr"},{"key":"B119-20230829","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1056\/NEJMc0806809","article-title":"Elimination of antibodies to recombinant enzyme in Pompe\u2019s disease","volume":"360","author":"Mendelsohn","year":"2009","journal-title":"N Engl J Med"},{"key":"B120-20230829","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1016\/j.ymgme.2012.01.019","article-title":"Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells","volume":"105","author":"Banugaria","year":"2012","journal-title":"Mol Genet Metab"},{"key":"B121-20230829","doi-asserted-by":"crossref","first-page":"e0134341","DOI":"10.1371\/journal.pone.0134341","article-title":"Oral migalastat HCl leads to greater systemic exposure and tissue levels of active \u03b1-galactosidase A in Fabry patients when co-administered with infused agalsidase","volume":"10","author":"Warnock","year":"2015","journal-title":"PLoS One"},{"key":"B122-20230829","doi-asserted-by":"crossref","first-page":"1630","DOI":"10.1021\/acs.bioconjchem.8b00133","article-title":"Development and analytical characterization of pegunigalsidase alfa, a chemically cross-linked plant recombinant human \u03b1-galactosidase-A for treatment of Fabry disease","volume":"29","author":"Ruderfer","year":"2018","journal-title":"Bioconjug Chem"},{"key":"B124-20230829","doi-asserted-by":"crossref","first-page":"e532","DOI":"10.1542\/peds.2003-0988-L","article-title":"Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease","volume":"114","author":"Hunley","year":"2004","journal-title":"Pediatrics"},{"key":"B125-20230829","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1681\/ASN.2013030290","article-title":"Allo-immune membranous nephropathy and recombinant aryl sulfatase replacement therapy: A need for tolerance induction therapy","volume":"25","author":"Debiec","year":"2014","journal-title":"J Am Soc Nephrol"},{"key":"B126-20230829","doi-asserted-by":"crossref","first-page":"1983","DOI":"10.1016\/S0140-6736(15)60731-0","article-title":"Pathophysiological advances in membranous nephropathy: Time for a shift in patient\u2019s care","volume":"385","author":"Ronco","year":"2015","journal-title":"Lancet"},{"key":"B127-20230829","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1681\/ASN.2014050502","article-title":"Identification of a major epitope recognized by PLA2R autoantibodies in primary membranous nephropathy","volume":"26","author":"Fresquet","year":"2015","journal-title":"J Am Soc Nephrol"}],"container-title":["Journal of the American Society of Nephrology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.ovid.com\/00001751-201809000-00005","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,7]],"date-time":"2026-08-07T19:30:48Z","timestamp":1786131048000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.ovid.com\/00001751-201809000-00005"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,8,9]]},"references-count":122,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2018]]}},"URL":"https:\/\/doi.org\/10.1681\/asn.2018030329","relation":{},"ISSN":["1046-6673","1533-3450"],"issn-type":[{"value":"1046-6673","type":"print"},{"value":"1533-3450","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,8,9]]}}}