{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T04:04:35Z","timestamp":1790395475075,"version":"4.1.0"},"reference-count":38,"publisher":"Oxford University Press (OUP)","issue":"8","license":[{"start":{"date-parts":[[2017,11,23]],"date-time":"2017-11-23T00:00:00Z","timestamp":1511395200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/about_us\/legal\/notices"}],"funder":[{"name":"Genzyme Europe"},{"DOI":"10.13039\/501100001659","name":"Deutsche Forschungsgemeinschaft","doi-asserted-by":"publisher","award":["Br1589\/8-2"],"award-info":[{"award-number":["Br1589\/8-2"]}],"id":[{"id":"10.13039\/501100001659","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002347","name":"Bundesministerium f\u00fcr Bildung und Forschung","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002347","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002347","name":"BMBF","doi-asserted-by":"publisher","award":["01EO1004"],"award-info":[{"award-number":["01EO1004"]}],"id":[{"id":"10.13039\/501100002347","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2018,8,1]]},"DOI":"10.1093\/ndt\/gfx319","type":"journal-article","created":{"date-parts":[[2017,10,18]],"date-time":"2017-10-18T07:09:45Z","timestamp":1508310585000},"page":"1362-1372","source":"Crossref","is-referenced-by-count":37,"title":["Fabry disease under enzyme replacement therapy\u2014new insights in efficacy of different dosages"],"prefix":"10.1093","volume":"33","author":[{"given":"Johannes","family":"Kr\u00e4mer","sequence":"first","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"},{"name":"Department of Pediatrics and Adolescent Medicine, University of Ulm, Ulm, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Malte","family":"Lenders","sequence":"additional","affiliation":[{"name":"Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center M\u00fcnster, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sima","family":"Canaan-K\u00fchl","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Nephrology, Charit\u00e9, Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Nordbeck","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nurcan","family":"\u00dc\u00e7eyler","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"},{"name":"Department of Neurology, University of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniela","family":"Blaschke","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Cardiology, Charit\u00e9, Campus Virchow-Klinikum, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Duning","sequence":"additional","affiliation":[{"name":"Department of Neurology, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefanie","family":"Reiermann","sequence":"additional","affiliation":[{"name":"Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center M\u00fcnster, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00f6rg","family":"Stypmann","sequence":"additional","affiliation":[{"name":"Department of Cardiovascular Medicine, Division of Cardiology, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan-Martin","family":"Brand","sequence":"additional","affiliation":[{"name":"Institute of Sports Medicine, Molecular Genetics of Cardiovascular Disease, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timo","family":"Gottschling","sequence":"additional","affiliation":[{"name":"Katharinen-Hospital Unna, Unna, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"St\u00f6rk","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Wanner","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Sommer","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"},{"name":"Department of Neurology, University of W\u00fcrzburg, W\u00fcrzburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eva","family":"Brand","sequence":"additional","affiliation":[{"name":"Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center M\u00fcnster, University Hospital M\u00fcnster, M\u00fcnster, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank","family":"Weidemann","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine I, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy (FAZIT), University Hospital and University of W\u00fcrzburg, W\u00fcrzburg, Germany"},{"name":"Katharinen-Hospital Unna, Unna, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2017,11,23]]},"reference":[{"key":"key\n\t\t\t\t20180801035943_gfx319-B1","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1056\/NEJM200107053450102","article-title":"Safety and efficacy of recombinant human alpha-galactosidase A\u2013replacement therapy in Fabry\u2019s disease","volume":"345","author":"Eng","year":"2001","journal-title":"N Engl J Med"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B2","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":"J Am Med Assoc"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B3","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1016\/j.ymgme.2010.11.155","article-title":"Expert opinion on temporarytreatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)","volume":"102","author":"Linthorst","year":"2011","journal-title":"Mol Genet Metab"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B4","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1186\/1750-1172-6-69","article-title":"Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients","volume":"6","author":"Smid","year":"2011","journal-title":"Orphanet J Rare Dis"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B5","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1007\/8904_2012_177","article-title":"Effects of switching from agalsidase Beta to agalsidase alfa in 10 patients with anderson-fabry disease","volume":"9","author":"Pisani","year":"2013","journal-title":"JIMD Rep"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B6","doi-asserted-by":"crossref","first-page":"779","DOI":"10.1038\/gim.2012.39","article-title":"Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagal)","volume":"14","author":"Tsuboi","year":"2012","journal-title":"Genet Med"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B7","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":"key\n\t\t\t\t20180801035943_gfx319-B8","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":"key\n\t\t\t\t20180801035943_gfx319-B9","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":"key\n\t\t\t\t20180801035943_gfx319-B10","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1136\/hrt.2006.104026","article-title":"Effects of enzyme replacement therapy on the cardiomyop- athy 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":"key\n\t\t\t\t20180801035943_gfx319-B11","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":"key\n\t\t\t\t20180801035943_gfx319-B12","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":"key\n\t\t\t\t20180801035943_gfx319-B13","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":"key\n\t\t\t\t20180801035943_gfx319-B14","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1111\/joim.12077","article-title":"Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications","volume":"274","author":"Weidemann","year":"2013","journal-title":"J Intern Med"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B15","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":"key\n\t\t\t\t20180801035943_gfx319-B16","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":"key\n\t\t\t\t20180801035943_gfx319-B17","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":"key\n\t\t\t\t20180801035943_gfx319-B18","doi-asserted-by":"crossref","first-page":"604","DOI":"10.7326\/0003-4819-150-9-200905050-00006","article-title":"A new equation to estimate glomerular filtration rate","volume":"150","author":"Levey","year":"2009","journal-title":"Ann Intern Med"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B19","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1056\/NEJMoa1114248","article-title":"Estimating glomerular filtration rate from serum creatinine and cystatin C","volume":"67","author":"Inker","year":"2012","journal-title":"N Engl J Med"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B20","doi-asserted-by":"crossref","first-page":"e47805","DOI":"10.1371\/journal.pone.0047805","article-title":"Long-term effect of antibodies against infused alphagalactosidase 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":"key\n\t\t\t\t20180801035943_gfx319-B21","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":"key\n\t\t\t\t20180801035943_gfx319-B22","doi-asserted-by":"crossref","first-page":"915","DOI":"10.1097\/AJP.0000000000000041","article-title":"Characterization of pain in Fabry disease","volume":"30","author":"\u00dc\u00e7eyler","year":"2014","journal-title":"Clin J Pain"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B23","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":"key\n\t\t\t\t20180801035943_gfx319-B24","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":"key\n\t\t\t\t20180801035943_gfx319-B25","doi-asserted-by":"crossref","first-page":"S139","DOI":"10.1097\/01.ASN.0000016683.73778.78","article-title":"Natural history and treatment of renal involvement in Fabry disease","volume":"13","author":"Branton","year":"2002","journal-title":"J Am Soc Nephrol"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B26","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1186\/1471-2369-15-72","article-title":"Fabry nephropathy: a review - how can we optimize the management of Fabry nephropathy?","volume":"15","author":"Waldek","year":"2014","journal-title":"BMC Nephrol"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B27","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1136\/heartjnl-2014-306278","article-title":"Cardiovascular outcomes in Fabry disease are linked to severity of chronic kidney disease","volume":"101","author":"Talbot","year":"2015","journal-title":"Heart"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B28","first-page":"2090","article-title":"Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease","volume":"32","author":"Lenders","year":"2017","journal-title":"Nephrol Dial Transplant"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B29","first-page":"1","article-title":"KDIGO 2012 clinical practice guideline for the evaluation and management of chronic kidney disease","volume":"3","author":"Kidney Disease: Improving Global Outcomes (KDIGO) CKD Work Group","year":"2013","journal-title":"Kidney Int Suppl"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B30","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1016\/j.ymgme.2014.01.014","article-title":"Outcomes of patients treated through the Canadian Fabry disease initiative","volume":"111","author":"Sirrs","year":"2014","journal-title":"Mol Genet Metab"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B31","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":"key\n\t\t\t\t20180801035943_gfx319-B32","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1038\/ki.2010.484","article-title":"Progressive podocyte injury and globotriaosylceramide (GL-3) accumulation in young patients with Fabry disease","volume":"79","author":"Najafian","year":"2011","journal-title":"Kidney Int"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B33","doi-asserted-by":"crossref","first-page":"S63","DOI":"10.1016\/j.ymgme.2015.12.307","article-title":"Comparison of integrated white blood cell alpha-galactosidase a activity exposure between every-other-day orally administered migalastat and biweekly infusions of agalsidase beta or agalsidase alfa","volume":"117","author":"Johnson","year":"2016","journal-title":"Mol Genet Metab"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B34","doi-asserted-by":"crossref","first-page":"572","DOI":"10.2337\/diacare.18.4.572","article-title":"Microalbuminuria and potential confounders. A review and some observations on variability of urinary albumin excretion","volume":"18","author":"Mogensen","year":"1995","journal-title":"Diabetes Care"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B35","doi-asserted-by":"crossref","first-page":"436","DOI":"10.1681\/ASN.2008030292","article-title":"First morning voids are more reliable than spot urine samples to assess microalbuminuria","volume":"20","author":"Witte","year":"2009","journal-title":"J Am Soc Nephrol"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B36","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":"key\n\t\t\t\t20180801035943_gfx319-B37","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1007\/8904_2015_422","article-title":"Effect and tolerability of agalsidase alfa in patients with Fabry disease who were treatment na\u00efve or formerly treated with agalsidase beta or agalsidase alfa","volume":"23","author":"Goker-Alpan","year":"2015","journal-title":"JIMD Reports"},{"key":"key\n\t\t\t\t20180801035943_gfx319-B38","doi-asserted-by":"crossref","first-page":"e0173358","DOI":"10.1371\/journal.pone.0173358","article-title":"Enzyme replacement therapy for Anderson\u2013Fabry 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"}],"container-title":["Nephrology Dialysis Transplantation"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/ndt\/article-pdf\/33\/8\/1362\/25198429\/gfx319.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,10,4]],"date-time":"2019-10-04T16:49:12Z","timestamp":1570207752000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/ndt\/article\/33\/8\/1362\/4653657"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,11,23]]},"references-count":38,"journal-issue":{"issue":"8","published-online":{"date-parts":[[2017,11,23]]},"published-print":{"date-parts":[[2018,8,1]]}},"URL":"https:\/\/doi.org\/10.1093\/ndt\/gfx319","relation":{},"ISSN":["0931-0509","1460-2385"],"issn-type":[{"value":"0931-0509","type":"print"},{"value":"1460-2385","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2018,8]]},"published":{"date-parts":[[2017,11,23]]}}}