{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T19:58:46Z","timestamp":1790711926181,"version":"4.1.0"},"reference-count":58,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2019,3,1]],"date-time":"2019-03-01T00:00:00Z","timestamp":1551398400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,3,1]],"date-time":"2019-03-01T00:00:00Z","timestamp":1551398400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2019,2,20]],"date-time":"2019-02-20T00:00:00Z","timestamp":1550620800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2019,3]]},"DOI":"10.1016\/j.ymgme.2018.04.007","type":"journal-article","created":{"date-parts":[[2018,4,26]],"date-time":"2018-04-26T03:43:42Z","timestamp":1524714222000},"page":"212-223","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":60,"title":["The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease \u2013 A systematic literature review by a European panel of experts"],"prefix":"10.1016","volume":"126","author":[{"given":"Marco","family":"Spada","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ralf","family":"Baron","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perry M.","family":"Elliott","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruno","family":"Falissard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Max J.","family":"Hilz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorenzo","family":"Monserrat","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camilla","family":"T\u00f8ndel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Tylki-Szyma\u0144ska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Wanner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dominique P.","family":"Germain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2018.04.007_bb0005","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1186\/1750-1172-5-30","article-title":"Fabry disease","volume":"5","author":"Germain","year":"2010","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0010","doi-asserted-by":"crossref","first-page":"769","DOI":"10.1093\/ndt\/gfp554","article-title":"End-stage renal disease in patients with Fabry disease: natural history data from the Fabry registry","volume":"25","author":"Ortiz","year":"2010","journal-title":"Nephrol. Dial. Transplant."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0015","doi-asserted-by":"crossref","first-page":"958","DOI":"10.1038\/gim.2013.53","article-title":"Analysis of left ventricular mass in untreated men and in men treated with agalsidase-\u03b2: data from the Fabry registry","volume":"15","author":"Germain","year":"2013","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0020","doi-asserted-by":"crossref","first-page":"788","DOI":"10.1161\/STROKEAHA.108.526293","article-title":"Stroke in Fabry disease frequently occurs before diagnosis and in the absence of other clinical events: natural history data from the Fabry registry","volume":"40","author":"Sims","year":"2009","journal-title":"Stroke"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0025","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1016\/j.ymgme.2007.09.013","article-title":"Females with Fabry disease frequently have major organ involvement: lessons from the Fabry registry","volume":"93","author":"Wilcox","year":"2008","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0030","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1203\/PDR.0b013e318183f132","article-title":"Characterization of Fabry disease in 352 pediatric patients in the Fabry registry","volume":"64","author":"Hopkin","year":"2008","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0035","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1016\/j.ijcard.2008.03.007","article-title":"Onset and progression of the Anderson-Fabry disease related cardiomyopathy","volume":"130","author":"Kampmann","year":"2008","journal-title":"Int. J. Cardiol."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0040","doi-asserted-by":"crossref","DOI":"10.3389\/fcvm.2016.00007","article-title":"Electrocardiographic changes and arrhythmia in Fabry disease","volume":"3","author":"Namdar","year":"2016","journal-title":"Front. Cardiovasc. Med."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0045","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1034\/j.1399-0004.2003.630107.x","article-title":"Fabry disease: a functional and anatomical study of cardiac manifestations in 20 hemizygous male patients","volume":"63","author":"Senechal","year":"2003","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0050","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1097\/01.gim.0000237866.70357.c6","article-title":"Fabry disease: guidelines for the evaluation and management of multi-organ system involvement","volume":"8","author":"Eng","year":"2006","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0055","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":"10.1016\/j.ymgme.2018.04.007_bb0060","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.ymgme.2018.04.007_bb0065","doi-asserted-by":"crossref","first-page":"641659","DOI":"10.1093\/qjmed\/hcq117","article-title":"Fabry disease: a review of current management strategies","volume":"103","author":"Mehta","year":"2010","journal-title":"QJM"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0070","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1093\/qjmed\/95.10.647","article-title":"New developments in the management of Anderson-Fabry disease","volume":"95","author":"Mehta","year":"2002","journal-title":"QJM"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0075","doi-asserted-by":"crossref","first-page":"21792205","DOI":"10.2165\/11318300-000000000-00000","article-title":"Enzyme replacement therapy for Fabry disease: a systematic review of available evidence","volume":"69","author":"Schaefer","year":"2009","journal-title":"Drugs"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0080","article-title":"Evaluation of the value of systematic reviews in rare diseases using Fabry disease as an exemplar","author":"Elliott","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0085","article-title":"The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease \u2013 a systematic literature review by a European panel of experts","author":"Germain","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0090","article-title":"The effect of enzyme replacement therapy on clinical outcomes in female patients with Fabry disease \u2013 a systematic literature review by a European panel of experts","author":"Germain","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0095","doi-asserted-by":"crossref","DOI":"10.1016\/j.ymgme.2018.06.004","article-title":"Expert consensus statement on therapeutic goals in Fabry disease","author":"Wanner","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0100","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1111\/j.1651-2227.2007.00029.x","article-title":"Enzyme replacement therapy with agalsidase alfa in children with Fabry disease","volume":"96","author":"Ramaswami","year":"2007","journal-title":"Acta Paediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0105","doi-asserted-by":"crossref","first-page":"924","DOI":"10.1542\/peds.2005-2895","article-title":"Enzyme-replacement therapy with agalsidase alfa in children with Fabry disease","volume":"118","author":"Ries","year":"2006","journal-title":"Pediatrics"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0110","doi-asserted-by":"crossref","first-page":"832","DOI":"10.1016\/j.jpeds.2009.11.007","article-title":"Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease","volume":"156","author":"Schiffmann","year":"2010","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0115","doi-asserted-by":"crossref","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":"10.1016\/j.ymgme.2018.04.007_bb0120","doi-asserted-by":"crossref","first-page":"1771","DOI":"10.2147\/DDDT.S102761","article-title":"An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are na\u00efve to enzyme replacement therapy","volume":"10","author":"Goker-Alpan","year":"2016","journal-title":"Drug Des. Dev. Ther."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0125","doi-asserted-by":"crossref","first-page":"1447","DOI":"10.1016\/j.cgh.2007.08.012","article-title":"Fabry Outcome Survey European investigators. 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":"10.1016\/j.ymgme.2018.04.007_bb0130","doi-asserted-by":"crossref","first-page":"605","DOI":"10.1111\/j.1651-2227.2010.02101.x","article-title":"Safety of agalsidase alfa in patients with Fabry disease under 7 years","volume":"100","author":"Ramaswami","year":"2011","journal-title":"Acta Paediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0135","doi-asserted-by":"crossref","first-page":"485","DOI":"10.1111\/j.1399-0004.2011.01671.x","article-title":"Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey","volume":"81","author":"Ramaswami","year":"2012","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0140","first-page":"53","article-title":"Early cardiac changes in children with Anderson-Fabry disease","volume":"11","author":"Havranek","year":"2013","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0145","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":"T\u00f8ndel","year":"2008","journal-title":"Am. J. Kidney Dis."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0150","doi-asserted-by":"crossref","first-page":"995","DOI":"10.1111\/j.1742-1241.2009.02323.x","article-title":"Beneficial effects of long-term enzyme replacement therapy in a child with Fabry disease","volume":"64","author":"Martin-Su\u00e1rez","year":"2010","journal-title":"Int. J. Clin. Pract."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0155","doi-asserted-by":"crossref","first-page":"1111","DOI":"10.1007\/s00431-013-2118-0","article-title":"A case of Fabry nephropathy with histological features of oligonephropathy","volume":"173","author":"Nishida","year":"2014","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0160","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1007\/s00431-003-1222-y","article-title":"Enzyme replacement therapy in an adolescent with Fabry disease","volume":"162","author":"Illsinger","year":"2003","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0165","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1016\/j.ymgme.2013.07.005","article-title":"Enzyme replacement therapy in two Japanese siblings with Fabry disease, and its effectiveness on angiokeratoma and neuropathic pain","volume":"110","author":"Furujo","year":"2013","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0170","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1159\/000369309","article-title":"Foot process effacement is an early marker of nephropathy in young classic Fabry patients without albuminuria","volume":"129","author":"T\u00f8ndel","year":"2015","journal-title":"Nephron"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0175","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.ymgme.2018.04.007_bb0180","doi-asserted-by":"crossref","first-page":"432","DOI":"10.1111\/j.1399-0004.2012.01947.x","article-title":"Fabry disease in children: agalsidase-beta enzyme replacement therapy","volume":"83","author":"Borgwardt","year":"2013","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0185","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1038\/jhg.2016.78","article-title":"Long-term enzyme replacement therapy for Fabry disease: efficacy and unmet needs in cardiac and renal outcomes","volume":"61","author":"Kim","year":"2016","journal-title":"J. Hum. Genet."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0190","first-page":"595","article-title":"Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease","volume":"163","author":"Mills","year":"2004","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0195","doi-asserted-by":"crossref","first-page":"679","DOI":"10.1007\/s00467-004-1462-8","article-title":"The co-existence of Fabry and celiac diseases: a case report","volume":"19","author":"T\u00fcmer","year":"2004","journal-title":"Pediatr. Nephrol."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0200","first-page":"20","article-title":"Fabry disease: a treatable lysosomal storage disorder","volume":"22","author":"Phadke","year":"2009","journal-title":"Natl Med. J. India"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0205","doi-asserted-by":"crossref","first-page":"207","DOI":"10.3344\/kjp.2010.23.3.207","article-title":"Early diagnosis of Fabry disease in a patient with toe tip pain","volume":"23","author":"Park","year":"2010","journal-title":"Korean J. Pain"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0210","first-page":"39","article-title":"Foot process effacement with normal urinalysis in classic Fabry disease","volume":"1","author":"Kanai","year":"2011","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0215","doi-asserted-by":"crossref","first-page":"1369","DOI":"10.1007\/s00467-016-3387-4","article-title":"Significant improvement in Fabry disease podocytopathy after 3 years of treatment with agalsidase beta","volume":"31","author":"Ito","year":"2016","journal-title":"Pediatr. Nephrol."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0220","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1007\/s00431-015-2646-x","article-title":"Surges in proteinuria are associated with plasma GL-3 elevations in a young patient with classic Fabry disease","volume":"175","author":"Kanai","year":"2016","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0225","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1111\/cge.12673","article-title":"Gastrointestinal involvement in Fabry disease. So important, yet often neglected","volume":"89","author":"Politei","year":"2016","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0230","doi-asserted-by":"crossref","first-page":"2013","DOI":"10.1021\/pr301200e","article-title":"The identification of new biomarkers for identifying and monitoring kidney disease and their translation into a rapid mass spectrometry-based test: evidence of presymptomatic kidney disease in pediatric Fabry and type-I diabetic patients","volume":"12","author":"Manwaring","year":"2013","journal-title":"J. Proteome Res."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0235","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":"10.1016\/j.ymgme.2018.04.007_bb0240","doi-asserted-by":"crossref","first-page":"969","DOI":"10.1007\/s10545-014-9717-4","article-title":"Long-term effectiveness of enzyme replacement therapy in Fabry disease: results from the NCS-LSD cohort study","volume":"37","author":"Anderson","year":"2014","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0245","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1002\/ajmg.b.32297","article-title":"The neurocognitive impact of Fabry disease on pediatric patients","volume":"168B","author":"Bugescu","year":"2015","journal-title":"Am. J. Med. Genet. B Neuropsychiatr. Genet."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0250","doi-asserted-by":"crossref","DOI":"10.1093\/ndt\/gfw094","article-title":"Reaccumulation of globotriaosylceramide in podocytes after agalsidase dose reduction in young Fabry patients","author":"Skrunes","year":"2016","journal-title":"Nephrol. Dial. Transplant."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0255","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1007\/s00467-009-1353-0","article-title":"A case of minimal change disease in a Fabry patient","volume":"25","author":"Zarate","year":"2010","journal-title":"Pediatr. Nephrol."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0260","first-page":"158","article-title":"Fabry's disease in a female, still an under-recognised disease","volume":"106","author":"Lynch","year":"2013","journal-title":"Ir. Med. J."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0265","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1186\/1756-0500-7-11","article-title":"De novo mutation in a male patient with Fabry disease: a case report","volume":"7","author":"Iemolo","year":"2014","journal-title":"BMC Res. Notes"},{"key":"10.1016\/j.ymgme.2018.04.007_bb0270","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1186\/1750-1172-8-47","article-title":"Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain","volume":"8","author":"Rombach","year":"2013","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0275","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":"10.1016\/j.ymgme.2018.04.007_bb0280","doi-asserted-by":"crossref","first-page":"1209","DOI":"10.1007\/s00216-005-3239-8","article-title":"Fast fingerprinting by MALDI-TOF mass spectrometry of urinary sediment glycosphingolipids in Fabry disease","volume":"382","author":"Touboul","year":"2005","journal-title":"Anal. Bioanal. Chem."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0285","doi-asserted-by":"crossref","first-page":"2812","DOI":"10.1073\/pnas.0712309105","article-title":"Elevated globotriaosylsphingosine is a hallmark of Fabry disease","volume":"105","author":"Aerts","year":"2008","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2018.04.007_bb0290","doi-asserted-by":"crossref","first-page":"262","DOI":"10.1136\/jmedgenet-2014-102872","article-title":"Plasma globotriaosylsphingosine in relation to phenotypes of Fabry disease","volume":"52","author":"Smid","year":"2015","journal-title":"J. Med. Genet."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719218301860?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719218301860?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T23:22:17Z","timestamp":1761088937000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719218301860"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,3]]},"references-count":58,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2019,3]]}},"alternative-id":["S1096719218301860"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2018.04.007","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2019,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease \u2013 A systematic literature review by a European panel of experts","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2018.04.007","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2019 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}