{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,26]],"date-time":"2026-08-26T17:22:45Z","timestamp":1787764965310,"version":"build-2784847793"},"reference-count":31,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2016,2,1]],"date-time":"2016-02-01T00:00:00Z","timestamp":1454284800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":["clinicalkey.jp","clinicalkey.com","clinicalkey.es","clinicalkey.com.au","clinicalkey.fr","jpeds.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["The Journal of Pediatrics"],"published-print":{"date-parts":[[2016,2]]},"DOI":"10.1016\/j.jpeds.2015.10.078","type":"journal-article","created":{"date-parts":[[2015,12,15]],"date-time":"2015-12-15T18:48:41Z","timestamp":1450205321000},"page":"174-180.e1","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":82,"special_numbering":"C","title":["Very Early Treatment for Infantile-Onset Pompe Disease Contributes to\u00a0Better Outcomes"],"prefix":"10.1016","volume":"169","author":[{"given":"Chia-Feng","family":"Yang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chen Chang","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hsuan-Chieh","family":"Liao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ling-Yi","family":"Huang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chuan-Chi","family":"Chiang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hui-Chen","family":"Ho","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chih-Jou","family":"Lai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tzu-Hung","family":"Chu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tsui-Feng","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ting-Rong","family":"Hsu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wen-Jue","family":"Soong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dau-Ming","family":"Niu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jpeds.2015.10.078_bib1","first-page":"304","article-title":"Concerning idiopathic hypertrophy of the heart","volume":"76","author":"Pompe","year":"1932","journal-title":"Ned Tijdschr Geneeskd"},{"key":"10.1016\/j.jpeds.2015.10.078_bib2","doi-asserted-by":"crossref","first-page":"S35","DOI":"10.1016\/j.jpeds.2004.01.053","article-title":"Pompe disease in infants and children","volume":"144","author":"Kishnani","year":"2004","journal-title":"J Pediatr"},{"key":"10.1016\/j.jpeds.2015.10.078_bib3","doi-asserted-by":"crossref","first-page":"332","DOI":"10.1542\/peds.112.2.332","article-title":"The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature","volume":"112","author":"van den Hout","year":"2003","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2015.10.078_bib4","doi-asserted-by":"crossref","first-page":"54","DOI":"10.1002\/ajmg.a.36197","article-title":"A large-scale nationwide newborn screening program for Pompe disease in Taiwan: towards effective diagnosis and treatment","volume":"164A","author":"Yang","year":"2014","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.jpeds.2015.10.078_bib5","first-page":"115","article-title":"Molecular study on the infantile form of Pompe disease in Chinese in Taiwan","volume":"37","author":"Lin","year":"1996","journal-title":"Zhonhua Min Guo Xiao Er Ke Yi Xue Hui Za Ahi"},{"key":"10.1016\/j.jpeds.2015.10.078_bib6","doi-asserted-by":"crossref","first-page":"e26","DOI":"10.4187\/respcare.03419","article-title":"Late-onset pompe disease with left-sided bronchomalacia","volume":"60","author":"Yang","year":"2015","journal-title":"Respir Care"},{"key":"10.1016\/j.jpeds.2015.10.078_bib7","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1016\/j.jpeds.2005.11.033","article-title":"A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease","volume":"148","author":"Kishnani","year":"2006","journal-title":"J Pediatr"},{"key":"10.1016\/j.jpeds.2015.10.078_bib8","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1016\/j.cca.2014.01.030","article-title":"Detecting multiple lysosomal storage diseases by tandem mass spectrometry\u2013a national newborn screening program in Taiwan","volume":"431","author":"Liao","year":"2014","journal-title":"Clin Chim Acta"},{"key":"10.1016\/j.jpeds.2015.10.078_bib9","doi-asserted-by":"crossref","first-page":"e39","DOI":"10.1542\/peds.2007-2222","article-title":"Early detection of Pompe disease by newborn screening is feasible: results from the Taiwan screening program","volume":"122","author":"Chien","year":"2008","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2015.10.078_bib10","doi-asserted-by":"crossref","first-page":"985","DOI":"10.1016\/j.jpeds.2014.10.068","article-title":"Long-term prognosis of patients with infantile-onset Pompe disease diagnosed by newborn screening and treated since birth","volume":"166","author":"Chien","year":"2015","journal-title":"J Pediatr"},{"key":"10.1016\/j.jpeds.2015.10.078_bib11","first-page":"S46","article-title":"Construction and validation of the Alberta Infant Motor Scale (AIMS)","volume":"83","author":"Piper","year":"1992","journal-title":"Can J Public Health"},{"key":"10.1016\/j.jpeds.2015.10.078_bib12","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1016\/j.ymgme.2011.10.012","article-title":"Early cognitive development in children with infantile Pompe disease","volume":"105","author":"Spiridigliozzi","year":"2012","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2015.10.078_bib13","doi-asserted-by":"crossref","first-page":"1512","DOI":"10.1212\/WNL.0b013e3182553c11","article-title":"Cognitive outcome of patients with classic infantile Pompe disease receiving enzyme therapy","volume":"78","author":"Ebbink","year":"2012","journal-title":"Neurology"},{"key":"10.1016\/j.jpeds.2015.10.078_bib14","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1007\/s10545-014-9707-6","article-title":"Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease","volume":"38","author":"van Gelder","year":"2015","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.jpeds.2015.10.078_bib15","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1097\/GIM.0b013e3182174703","article-title":"The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease","volume":"13","author":"Banugaria","year":"2011","journal-title":"Genet Med"},{"key":"10.1016\/j.jpeds.2015.10.078_bib16","doi-asserted-by":"crossref","first-page":"190","DOI":"10.1016\/j.ymgme.2009.03.004","article-title":"High frequency of acid alpha-glucosidase pseudodeficiency complicates newborn screening for glycogen storage disease type II in the Japanese population","volume":"97","author":"Kumamoto","year":"2009","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2015.10.078_bib17","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":"10.1016\/j.jpeds.2015.10.078_bib18","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1016\/j.ymgme.2011.09.002","article-title":"Newborn screening for Pompe disease in Japan","volume":"104","author":"Oda","year":"2011","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2015.10.078_bib19","doi-asserted-by":"crossref","first-page":"1327","DOI":"10.1542\/peds.2007-0388","article-title":"Newborn screening for Pompe disease: synthesis of the evidence and development of screening recommendations","volume":"120","author":"Kemper","year":"2007","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2015.10.078_bib20","doi-asserted-by":"crossref","first-page":"775","DOI":"10.1016\/j.nmd.2010.07.277","article-title":"Effect of enzyme therapy in unvenile patients with Pompe disease: a three-year open-label study","volume":"20","author":"van Capelle","year":"2010","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.jpeds.2015.10.078_bib21","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.recesp.2011.03.025","article-title":"When should we start enzyme replacement therapy for infant Pompe disease with severe cardiomyopathy?","volume":"65","author":"Bonilla-Palomas","year":"2012","journal-title":"Rev Esp Cardiol"},{"key":"10.1016\/j.jpeds.2015.10.078_bib22","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1002\/ajmg.c.31320","article-title":"Assessing disease severity in Pompe disease: the roles of a urinary glucose tetrasaccharide biomaker and imaging techniques","volume":"160C","author":"Young","year":"2012","journal-title":"Am J Med Genet C Semin Med Genet"},{"key":"10.1016\/j.jpeds.2015.10.078_bib23","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1002\/ajmg.c.31321","article-title":"Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations","volume":"160C","author":"Case","year":"2012","journal-title":"Am J Med Genet C Semin Med Genet"},{"key":"10.1016\/j.jpeds.2015.10.078_bib24","doi-asserted-by":"crossref","first-page":"e1116","DOI":"10.1542\/peds.2008-3667","article-title":"Pompe disease in infants: improving the prognosis by newborn screening and early treatment","volume":"124","author":"Chien","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2015.10.078_bib25","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1016\/j.ymgme.2009.08.003","article-title":"Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants","volume":"99","author":"Kishnani","year":"2010","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2015.10.078_bib26","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":"10.1016\/j.jpeds.2015.10.078_bib27","doi-asserted-by":"crossref","first-page":"1396","DOI":"10.1056\/NEJMoa0909859","article-title":"A randomized study of alglucosidase alfa in late-onset Pompe's disease","volume":"362","author":"van der Ploeg","year":"2010","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jpeds.2015.10.078_bib28","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1016\/j.ymgme.2012.04.027","article-title":"The impact of antibodies in late-onset Pompe disease: a case series and literature review","volume":"106","author":"Patel","year":"2012","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2015.10.078_bib29","doi-asserted-by":"crossref","first-page":"268","DOI":"10.1006\/mgme.1999.2894","article-title":"Immune response to enzyme replacement therapy in lysosomal storage disorder patients and animal models","volume":"68","author":"Brooks","year":"1999","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2015.10.078_bib30","doi-asserted-by":"crossref","first-page":"60ra89","DOI":"10.1126\/scitranslmed.3001380","article-title":"Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I","volume":"2","author":"Dierenfeld","year":"2010","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.jpeds.2015.10.078_bib31","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1126\/science.1071059","article-title":"The danger model: a renewed sense of self","volume":"296","author":"Matzinger","year":"2002","journal-title":"Science"}],"container-title":["The Journal of Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022347615013177?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022347615013177?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,9,16]],"date-time":"2018-09-16T20:25:59Z","timestamp":1537129559000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0022347615013177"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,2]]},"references-count":31,"alternative-id":["S0022347615013177"],"URL":"https:\/\/doi.org\/10.1016\/j.jpeds.2015.10.078","relation":{},"ISSN":["0022-3476"],"issn-type":[{"value":"0022-3476","type":"print"}],"subject":[],"published":{"date-parts":[[2016,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Very Early Treatment for Infantile-Onset Pompe Disease Contributes to\u00a0Better Outcomes","name":"articletitle","label":"Article Title"},{"value":"The Journal of Pediatrics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jpeds.2015.10.078","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2016 Published by Mosby, Inc.","name":"copyright","label":"Copyright"}]}}