{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T06:58:11Z","timestamp":1761807491101,"version":"build-2065373602"},"reference-count":34,"publisher":"Elsevier BV","issue":"10","license":[{"start":{"date-parts":[[2009,10,1]],"date-time":"2009-10-01T00:00:00Z","timestamp":1254355200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2009,10,1]],"date-time":"2009-10-01T00:00:00Z","timestamp":1254355200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"content-domain":{"domain":["clinicalkey.fr","em-consulte.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["La Revue de M\u00e9decine Interne"],"published-print":{"date-parts":[[2009,10]]},"DOI":"10.1016\/j.revmed.2009.04.003","type":"journal-article","created":{"date-parts":[[2009,6,13]],"date-time":"2009-06-13T04:38:52Z","timestamp":1244867932000},"page":"920-929","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":3,"title":["Efficacit\u00e9 clinique de l\u2019enzymoth\u00e9rapie dans la maladie de Fabry. Analyse critique"],"prefix":"10.1016","volume":"30","author":[{"given":"O.","family":"Lidove","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"T.","family":"Papo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.revmed.2009.04.003_bib1","doi-asserted-by":"crossref","first-page":"289","DOI":"10.2174\/187152506778520718","article-title":"The role of ceramide trihexoside (globotriaosylceramide) in the diagnosis and follow-up of the efficacy of treatment of Fabry disease: a review of the literature","volume":"4","author":"Bekri","year":"2006","journal-title":"Cardiovasc Hematol Agents Med Chem"},{"key":"10.1016\/j.revmed.2009.04.003_bib2","doi-asserted-by":"crossref","first-page":"1084","DOI":"10.1016\/j.lpm.2007.01.006","article-title":"Fabry disease: proposed guidelines from a French expert group for its diagnosis, treatment and follow-up","volume":"36","author":"Lidove","year":"2007","journal-title":"Presse Med"},{"key":"10.1016\/j.revmed.2009.04.003_bib3","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":"Lee","year":"2003","journal-title":"Glycobiology"},{"key":"10.1016\/j.revmed.2009.04.003_bib4","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":"Desnick","year":"2004","journal-title":"Expert Opin Biol Ther"},{"key":"10.1016\/j.revmed.2009.04.003_bib5","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1086\/318809","article-title":"A phase 1\/2 clinical trial of enzyme replacement in fabry disease: pharmacokinetic, substrate clearance, and safety studies","volume":"68","author":"Eng","year":"2001","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.revmed.2009.04.003_bib6","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's disease","volume":"345","author":"Eng","year":"2001","journal-title":"N Engl J Med"},{"key":"10.1016\/j.revmed.2009.04.003_bib7","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":"10.1016\/j.revmed.2009.04.003_bib8","doi-asserted-by":"crossref","first-page":"1547","DOI":"10.1681\/ASN.2006080816","article-title":"Sustained, long-term renal stabilization after 54\u00a0months of agalsidase beta therapy in patients with Fabry disease","volume":"18","author":"Germain","year":"2007","journal-title":"J Am Soc Nephrol"},{"key":"10.1016\/j.revmed.2009.04.003_bib9","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1073\/pnas.97.1.365","article-title":"Infusion of alpha-galactosidase A reduces tissue globotriaosylceramide storage in patients with Fabry disease","volume":"97","author":"Schiffmann","year":"2000","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.revmed.2009.04.003_bib10","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":"10.1016\/j.revmed.2009.04.003_bib11","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":"Schiffmann","year":"2003","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.revmed.2009.04.003_bib12","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"},{"key":"10.1016\/j.revmed.2009.04.003_bib13","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1093\/ndt\/gfi152","article-title":"Long-term therapy with agalsidase alpha 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":"10.1016\/j.revmed.2009.04.003_bib14","doi-asserted-by":"crossref","first-page":"2131","DOI":"10.1038\/sj.ki.5000410","article-title":"The podocyte's response to injury: role in proteinuria and glomerulosclerosis","volume":"69","author":"Shankland","year":"2006","journal-title":"Kidney Int"},{"key":"10.1016\/j.revmed.2009.04.003_bib15","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":"10.1016\/j.revmed.2009.04.003_bib16","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1093\/ejechocard\/jen129","article-title":"Anderson-Fabry disease: long-term echocardiographic follow-up under enzyme replacement therapy","volume":"9","author":"Kovacevic-Preradovic","year":"2008","journal-title":"Eur J Echocardiogr"},{"key":"10.1016\/j.revmed.2009.04.003_bib17","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1161\/01.CIR.54.5.818","article-title":"Cardiac valvular anomalies in Fabry disease. Clinical, morphologic, and biochemical studies","volume":"54","author":"Desnick","year":"1976","journal-title":"Circulation"},{"key":"10.1016\/j.revmed.2009.04.003_bib18","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 alpha","volume":"94","author":"Hughes","year":"2008","journal-title":"Heart"},{"key":"10.1016\/j.revmed.2009.04.003_bib19","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1023\/B:BOLI.0000028794.04349.91","article-title":"CNS involvement in Fabry disease: clinical and imaging studies before and after 12\u00a0months of enzyme replacement therapy","volume":"27","author":"Jardim","year":"2004","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.revmed.2009.04.003_bib20","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":"10.1016\/j.revmed.2009.04.003_bib21","first-page":"77","article-title":"Enzyme replacement therapy and renal function in 201\u00a0patients with Fabry disease","volume":"66","author":"Schwarting","year":"2006","journal-title":"Clin Nephrol"},{"key":"10.1016\/j.revmed.2009.04.003_bib22","doi-asserted-by":"crossref","first-page":"2609","DOI":"10.1681\/ASN.2006121400","article-title":"Antiproteinuric therapy and fabry nephropathy: sustained reduction of proteinuria in patients receiving enzyme replacement therapy with agalsidase-beta","volume":"18","author":"Tahir","year":"2007","journal-title":"J Am Soc Nephrol"},{"key":"10.1016\/j.revmed.2009.04.003_bib23","doi-asserted-by":"crossref","first-page":"924","DOI":"10.1542\/peds.2005-2895","article-title":"Enzyme-replacement therapy with agalsidase alpha in children with Fabry disease","volume":"118","author":"Ries","year":"2006","journal-title":"Pediatrics"},{"key":"10.1016\/j.revmed.2009.04.003_bib24","doi-asserted-by":"crossref","first-page":"767","DOI":"10.1053\/j.ajkd.2007.12.032","article-title":"Renal biopsy findings in children and adolescents with Fabry disease and minimal albuminuria","volume":"51","author":"Tondel","year":"2008","journal-title":"Am J Kidney Dis"},{"key":"10.1016\/j.revmed.2009.04.003_bib25","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":"10.1016\/j.revmed.2009.04.003_bib26","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1111\/j.1365-2362.2004.01424.x","article-title":"Fabry disease: overall effects of agalsidase alpha treatment","volume":"34","author":"Beck","year":"2004","journal-title":"Eur J Clin Invest"},{"key":"10.1016\/j.revmed.2009.04.003_bib27","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":"10.1016\/j.revmed.2009.04.003_bib28","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":"10.1016\/j.revmed.2009.04.003_bib29","doi-asserted-by":"crossref","first-page":"e598","DOI":"10.1371\/journal.pone.0000598","article-title":"Treatment of Fabry disease: outcome of a comparative trial with agalsidase alpha or beta at a dose of 0.2 mg\/kg","volume":"2","author":"Vedder","year":"2007","journal-title":"PLoS ONE"},{"key":"10.1016\/j.revmed.2009.04.003_bib30","doi-asserted-by":"crossref","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","author":"Vedder","year":"2008","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.revmed.2009.04.003_bib31","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":"10.1016\/j.revmed.2009.04.003_bib32","doi-asserted-by":"crossref","first-page":"1467","DOI":"10.1517\/13543784.11.10.1467","article-title":"Fabry disease: recent advances in enzyme replacement therapy","volume":"11","author":"Germain","year":"2002","journal-title":"Expert Opin Investig Drugs"},{"key":"10.1016\/j.revmed.2009.04.003_bib33","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":"10.1016\/j.revmed.2009.04.003_bib34","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":"Whitfield","year":"2005","journal-title":"J Inherit Metab Dis"}],"container-title":["La Revue de M\u00e9decine Interne"],"original-title":[],"language":"fr","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0248866309005852?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0248866309005852?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T16:24:07Z","timestamp":1759335847000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0248866309005852"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,10]]},"references-count":34,"journal-issue":{"issue":"10","published-print":{"date-parts":[[2009,10]]}},"alternative-id":["S0248866309005852"],"URL":"https:\/\/doi.org\/10.1016\/j.revmed.2009.04.003","relation":{},"ISSN":["0248-8663"],"issn-type":[{"type":"print","value":"0248-8663"}],"subject":[],"published":{"date-parts":[[2009,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Efficacit\u00e9 clinique de l\u2019enzymoth\u00e9rapie dans la maladie de Fabry. Analyse critique","name":"articletitle","label":"Article Title"},{"value":"La Revue de M\u00e9decine Interne","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.revmed.2009.04.003","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2009 Soci\u00e9t\u00e9 nationale fran\u00e7aise de m\u00e9decine interne (SNFMI). Published by Elsevier Masson SAS All rights reserved.","name":"copyright","label":"Copyright"}]}}