{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,5]],"date-time":"2026-11-05T23:50:43Z","timestamp":1793922643675,"version":"4.8.0"},"reference-count":39,"publisher":"Public Library of Science (PLoS)","issue":"7","license":[{"start":{"date-parts":[[2007,7,11]],"date-time":"2007-07-11T00:00:00Z","timestamp":1184112000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0000598","type":"journal-article","created":{"date-parts":[[2007,7,10]],"date-time":"2007-07-10T20:35:54Z","timestamp":1184099754000},"page":"e598","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":159,"title":["Treatment of Fabry Disease: Outcome of a Comparative Trial with Agalsidase Alfa or Beta at a Dose of 0.2 mg\/kg"],"prefix":"10.1371","volume":"2","clinical-trial-number":[{"clinical-trial-number":"isrctn45178534","registry":"10.18810\/isrctn"}],"author":[{"given":"Anouk C.","family":"Vedder","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabor E.","family":"Linthorst","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gunnar","family":"Houge","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannna E.M.","family":"Groener","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Els E.","family":"Ormel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Berto J.","family":"Bouma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Johannes M.F.G.","family":"Aerts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Asle","family":"Hirth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla E.M.","family":"Hollak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2007,7,11]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"1163","DOI":"10.1056\/NEJM196705252762101","article-title":"Enzymatic defect in Fabry&apos;s disease. Ceramidetrihexosidase deficiency.","volume":"276","author":"RO Brady","year":"1967","journal-title":"N Engl J Med"},{"key":"ref2","doi-asserted-by":"crossref","first-page":"1268","DOI":"10.1126\/science.167.3922.1268","article-title":"Fabry&apos;s disease: alpha-galactosidase deficiency.","volume":"167","author":"JA Kint","year":"1970","journal-title":"Science"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"682","DOI":"10.1136\/jmg.33.8.682","article-title":"Uneven X inactivation in a female monozygotic twin pair with Fabry disease and discordant expression of a novel mutation in the alpha-galactosidase A gene.","volume":"33","author":"I Redonnet-Vernhet","year":"1996","journal-title":"J Med Genet"},{"key":"ref4","first-page":"3733","article-title":"\u03b1-Galactosidase A Deficiency: Fabry disease.","author":"RJ Desnick","year":"2001"},{"key":"ref5","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":"KD MacDermot","year":"2001","journal-title":"J Med Genet"},{"key":"ref6","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1056\/NEJM200107053450102","article-title":"Safety and efficacy of recombinant human \u03b1-galactosidase A replacement therapy in Fabry&apos;s disease.","volume":"345","author":"CM Eng","year":"2001","journal-title":"N Engl J Med"},{"key":"ref7","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":"R Schiffmann","year":"2001","journal-title":"JAMA"},{"key":"ref8","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":"M Beck","year":"2004","journal-title":"Eur J Clin Invest"},{"key":"ref9","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1086\/422366","article-title":"Long-Term Safety and Efficacy of Enzyme Replacement Therapyfor Fabry Disease.","volume":"75","author":"WR Wilcox","year":"2004","journal-title":"Am J Hum Genet"},{"key":"ref10","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":"F Weidemann","year":"2003","journal-title":"Circulation"},{"key":"ref11","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1086\/345309","article-title":"Recombinant enzyme therapy for Fabry disease. Absence of editing of human \u03b1-Galactosidase A mRNA.","volume":"72","author":"D Blom","year":"2003","journal-title":"Am J Hum Genet"},{"key":"ref12","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1093\/glycob\/cwg034","article-title":"A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease.","volume":"13","author":"K Lee","year":"2003","journal-title":"Glycobiology"},{"key":"ref13","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1007\/s10038-005-0342-9","article-title":"Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice.","volume":"51","author":"H Sakuraba","year":"2005","journal-title":"J Hum Genet"},{"key":"ref14","year":"2001"},{"key":"ref15","doi-asserted-by":"crossref","first-page":"601","DOI":"10.1016\/S0140-6736(01)05816-0","article-title":"Enzyme-replacement therapy for Anderson-Fabry disease.","volume":"358","author":"GM Pastores","year":"2001","journal-title":"The Lancet"},{"key":"ref16","year":"2002"},{"key":"ref17","doi-asserted-by":"crossref","first-page":"956","DOI":"10.1016\/0002-9149(87)91133-7","article-title":"Echocardiographic criteria for left ventricular hypertrophy: the Framingham Heart Study.","volume":"59","author":"D Levy","year":"1987","journal-title":"Am J Cardiol"},{"key":"ref18","doi-asserted-by":"crossref","first-page":"1625","DOI":"10.1016\/S0735-1097(99)00396-4","article-title":"Reliability of echocardiographic assessment of left ventricular structure and function: the PRESERVE study. Prospective Randomized Study Evaluating Regression of Ventricular Enlargement.","volume":"34","author":"V Palmieri","year":"1999","journal-title":"J Am Coll Cardiol"},{"key":"ref19","doi-asserted-by":"crossref","first-page":"567","DOI":"10.1681\/ASN.V74567","article-title":"Precision of glomerular filtration rate determinations for long-term slope calculations is improved by simultaneous infusion of 125I- iothalamate and 131I-hippuran.","volume":"7","author":"AJ Apperloo","year":"1996","journal-title":"J Am Soc Nephrol"},{"key":"ref20","doi-asserted-by":"crossref","first-page":"310","DOI":"10.1681\/ASN.V92310","article-title":"Precision of plasma clearance of iohexol for estimation of GFR in patients with renal disease.","volume":"9","author":"F Gaspari","year":"1998","journal-title":"J Am Soc Nephrol"},{"key":"ref21","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":"C Whybra","year":"2004","journal-title":"Clin Genet"},{"key":"ref22","doi-asserted-by":"crossref","first-page":"1139","DOI":"10.1212\/WNL.32.10.1139","article-title":"Fabry disease: detection of heterozygotes by examination of glycolipids in urinary sediment.","volume":"32","author":"WJ Cable","year":"1982","journal-title":"Neurology"},{"key":"ref23","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":"GE Linthorst","year":"2004","journal-title":"Kidney Int"},{"key":"ref24","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1016\/S0021-9258(18)64849-5","article-title":"A Simple Method for the Isolation and Purification of Total Lipids from Animal Tissues.","volume":"226","author":"J Folch","year":"1957","journal-title":"J Biol Chem"},{"key":"ref25","doi-asserted-by":"crossref","first-page":"573","DOI":"10.1093\/oxfordjournals.jbchem.a021452","article-title":"Rapid method of preparation of lysoglycosphingolipids and their confirmation by delayed extraction matrix-assisted laser desorption ionization time-of-flight mass spectrometry.","volume":"120","author":"T Taketomi","year":"1996","journal-title":"J Biochem (Tokyo)"},{"key":"ref26","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1016\/S0140-6736(03)12357-4","article-title":"Enzyme replacement in Anderson-Fabry disease.","volume":"361","author":"BA Bengtsson","year":"2003","journal-title":"Lancet"},{"key":"ref27","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1097\/01.GIM.0000069509.57929.CD","article-title":"Comparative evaluation of alpha-galactosidase A infusions for treatment of Fabry disease.","volume":"5","author":"RJ Hopkin","year":"2003","journal-title":"Genet Med"},{"key":"ref28","doi-asserted-by":"crossref","first-page":"1167","DOI":"10.1517\/14712598.4.7.1167","article-title":"Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval.","volume":"4","author":"RJ Desnick","year":"2004","journal-title":"Expert Opin Biol Ther"},{"key":"ref29","first-page":"354","article-title":"Enzyme replacement therapy in Fabry&apos;s disease: recent advances and clinical applications.","volume":"17","author":"R Mignani","year":"2004","journal-title":"J Nephrol"},{"key":"ref30","doi-asserted-by":"crossref","first-page":"703","DOI":"10.1002\/mus.10497","article-title":"Enzyme replacement therapy improves peripheral nerve and sweat function in Fabry disease.","volume":"28","author":"R Schiffmann","year":"2003","journal-title":"Muscle Nerve"},{"key":"ref31","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":"R Schiffmann","year":"2006","journal-title":"Nephrol Dial Transplant"},{"key":"ref32","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":"B Hoffmann","year":"2005","journal-title":"J Med Genet"},{"key":"ref33","first-page":"77","article-title":"Enzyme replacement therapy and renal function in 201 patients with Fabry disease.","volume":"66","author":"A Schwarting","year":"2006","journal-title":"Clin Nephrol"},{"key":"ref34","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&apos;s disease.","volume":"66","author":"L Spinelli","year":"2004","journal-title":"Clin Genet"},{"key":"ref35","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":"F Breunig","year":"2006","journal-title":"Kidney Int"},{"key":"ref36","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":"M Banikazemi","year":"2007","journal-title":"Ann Intern Med"},{"key":"ref37","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1007\/s10545-005-4415-x","article-title":"Monitoring enzyme replacement therapy in Fabry disease-Role of urine globotriaosylceramide.","volume":"28","author":"PD Whitfield","year":"2005","journal-title":"J Inherit Metab Dis"},{"key":"ref38","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":"R Schiffmann","year":"2005","journal-title":"Nephrol Dial Transplant"},{"key":"ref39","doi-asserted-by":"crossref","first-page":"e344","DOI":"10.1542\/peds.2004-1678","article-title":"Pediatric Fabry Disease.","volume":"115","author":"M Ries","year":"2005","journal-title":"Pediatrics"}],"container-title":["PLoS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0000598","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,8,18]],"date-time":"2021-08-18T20:36:13Z","timestamp":1629318973000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0000598"}},"subtitle":[],"editor":[{"given":"Raphael","family":"Schiffmann","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2007,7,11]]},"references-count":39,"journal-issue":{"issue":"7","published-online":{"date-parts":[[2007,7,11]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0000598","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2007,7,11]]}}}