{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,2]],"date-time":"2026-04-02T15:02:22Z","timestamp":1775142142191,"version":"3.50.1"},"update-to":[{"DOI":"10.1002\/14651858.cd011539","type":"new_version","label":"New version","source":"publisher","updated":{"date-parts":[[2017,11,20]],"date-time":"2017-11-20T00:00:00Z","timestamp":1511136000000}}],"reference-count":45,"publisher":"Wiley","issue":"12","content-domain":{"domain":["wiley.com","cochranelibrary.com"],"crossmark-restriction":true},"short-container-title":[],"DOI":"10.1002\/14651858.cd011539.pub2","type":"journal-article","created":{"date-parts":[[2017,11,20]],"date-time":"2017-11-20T15:53:40Z","timestamp":1511193220000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":20,"title":["Enzyme replacement therapy for infantile-onset Pompe disease"],"prefix":"10.1002","volume":"2017","author":[{"given":"Min","family":"Chen","sequence":"first","affiliation":[{"name":"West China Second University Hospital, Sichuan University; Department of Pharmacy; No. 20, Section 4, Renmin South Road Chengdu China 610041"},{"name":"Sichuan University, Ministry of Education; Key Laboratory of Birth Defects and Related Diseases of Women and Children; Chengdu China"},{"name":"West China Second University Hospital, Sichuan University; Evidence-Based Pharmacy Center; Chengdu China"},{"name":"Sichuan University; Department of Obstetrics and Gynecology, West China Second University Hospital; Chengdu China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lingli","family":"Zhang","sequence":"additional","affiliation":[{"name":"West China Second University Hospital, Sichuan University; Department of Pharmacy; No. 20, Section 4, Renmin South Road Chengdu China 610041"},{"name":"Sichuan University, Ministry of Education; Key Laboratory of Birth Defects and Related Diseases of Women and Children; Chengdu China"},{"name":"West China Second University Hospital, Sichuan University; Evidence-Based Pharmacy Center; Chengdu China"},{"name":"Sichuan University; Department of Obstetrics and Gynecology, West China Second University Hospital; Chengdu China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shuyan","family":"Quan","sequence":"additional","affiliation":[{"name":"West China Second University Hospital, Sichuan University; Department of Pharmacy; No. 20, Section 4, Renmin South Road Chengdu China 610041"},{"name":"Sichuan University, Ministry of Education; Key Laboratory of Birth Defects and Related Diseases of Women and Children; Chengdu China"},{"name":"West China Second University Hospital, Sichuan University; Evidence-Based Pharmacy Center; Chengdu China"},{"name":"Sichuan University; Department of Obstetrics and Gynecology, West China Second University Hospital; Chengdu China"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2017,11,20]]},"reference":[{"issue":"Suppl 1","key":"10.1002\/14651858.CD011539.pub2-BIB0001|cit1","first-page":"195","article-title":"Enzyme replacement therapy (ERT) with recombinant human acid alpha glucosidase (rhGAA) in infantile onset Pompe disease (IOPD)","volume":"28","author":"Kishnani","year":"2005","journal-title":"Journal of Inherited Metabolic Disease"},{"issue":"9-10","key":"10.1002\/14651858.CD011539.pub2-BIB0002|cit2","first-page":"712","article-title":"Enzyme replacement therapy with recombinant human acid alpha glucosidase (rhGAA) in infantile onset Pompe disease (IOPD)","volume":"15","author":"Kishnani","year":"2005","journal-title":"Neuromuscular Disorders : NMD"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0003|cit3","doi-asserted-by":"crossref","unstructured":"Kishnani PS Corzo D Leslie ND Gruskin D Van der Ploeg A Clancy JP Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease Pediatric Research 2009 329 35","DOI":"10.1203\/PDR.0b013e3181b24e94"},{"issue":"2","key":"10.1002\/14651858.CD011539.pub2-BIB0004|cit4","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1212\/01.wnl.0000251268.41188.04","article-title":"Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease","volume":"68","author":"Kishnani","year":"2007","journal-title":"Neurology"},{"issue":"4","key":"10.1002\/14651858.CD011539.pub2-BIB0005|cit5","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1016\/j.nmd.2014.12.004","article-title":"Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease","volume":"25","author":"Case","year":"2015","journal-title":"Neuromuscular Disorders"},{"issue":"6","key":"10.1002\/14651858.CD011539.pub2-BIB0006|cit6","doi-asserted-by":"crossref","first-page":"747","DOI":"10.1007\/s10545-010-9206-3","article-title":"Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience","volume":"33","author":"Chakrapani","year":"2010","journal-title":"Journal of Inherited Metabolic Disease"},{"issue":"11","key":"10.1002\/14651858.CD011539.pub2-BIB0007|cit7","doi-asserted-by":"crossref","first-page":"930","DOI":"10.1016\/j.cardfail.2011.07.011","article-title":"Left ventricular geometry, global function, and dyssynchrony in infants and children with Pompe cardiomyopathy undergoing enzyme replacement therapy","volume":"17","author":"Chen","year":"2011","journal-title":"Journal of Cardiac Failure"},{"issue":"5","key":"10.1002\/14651858.CD011539.pub2-BIB0008|cit8","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1097\/01.gim.0000217786.79173.a8","article-title":"Ambulatory electrocardiogram analysis in infants treated with recombinant human acid \u03b1-glucosidase enzyme replacement therapy for Pompe disease","volume":"8","author":"Cook","year":"2006","journal-title":"Genetics in Medicine"},{"issue":"1","key":"10.1002\/14651858.CD011539.pub2-BIB0009|cit9","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.jpeds.2006.02.035","article-title":"Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease","volume":"149","author":"Kishnani","year":"2006","journal-title":"Journal of Pediatrics"},{"issue":"9-10","key":"10.1002\/14651858.CD011539.pub2-BIB0010|cit10","doi-asserted-by":"crossref","first-page":"832","DOI":"10.1016\/j.nmd.2008.06.379","article-title":"Safety and efficacy results from a randomized, double-blind, placebo-controlled study of alglucosidase alfa for the treatment of Pompe disease in juveniles and adults","volume":"18","author":"Lafor\u00eat","year":"2008","journal-title":"Neuromuscular Disorders : NMD"},{"issue":"Suppl 3","key":"10.1002\/14651858.CD011539.pub2-BIB0011|cit11","first-page":"412-3, Abstract","article-title":"Safety and efficacy results from a randomized, double-blind, placebo-controlled study of alglucosidase alpha for the treatment of Pompe's disease in juveniles and adults","volume":"15","author":"Ploeg","year":"2008","journal-title":"European Journal of Neurology"},{"issue":"6","key":"10.1002\/14651858.CD011539.pub2-BIB0012|cit12","doi-asserted-by":"crossref","first-page":"1033","DOI":"10.1007\/s00246-008-9267-3","article-title":"Cardiac remodeling after enzyme replacement therapy with acid \u03b1-glucosidase for infants with Pompe disease","volume":"29","author":"Levine","year":"2008","journal-title":"Pediatric Cardiology"},{"issue":"3","key":"10.1002\/14651858.CD011539.pub2-BIB0013|cit13","doi-asserted-by":"crossref","first-page":"210","DOI":"10.1097\/GIM.0b013e31819d0996","article-title":"Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease","volume":"11","author":"Nicolino","year":"2009","journal-title":"Genetics in Medicine"},{"issue":"1","key":"10.1002\/14651858.CD011539.pub2-BIB0014|cit14","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1186\/1750-1172-8-90","article-title":"Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy","volume":"8","author":"Prater","year":"2013","journal-title":"Orphanet Journal of Rare Diseases"},{"issue":"Suppl 2","key":"10.1002\/14651858.CD011539.pub2-BIB0015|cit15","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1186\/1471-2474-14-S2-P19","article-title":"A higher dose of enzyme therapy in patients with classic infantile Pompe disease seems to improve ventilator-free survival and motor function","volume":"14","author":"Gelder","year":"2013","journal-title":"BMC Musculoskeletal Disorders"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0016|cit16","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1007\/8904_2012_192","article-title":"CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy","volume":"9","author":"Al Khallaf","year":"2013","journal-title":"JIMD Reports"},{"issue":"2","key":"10.1002\/14651858.CD011539.pub2-BIB0017|cit17","first-page":"132","article-title":"Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I\/II clinical trial","volume":"3","author":"Amalfitano","year":"2001","journal-title":"Genetics in Medicine"},{"issue":"8","key":"10.1002\/14651858.CD011539.pub2-BIB0018|cit18","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":"Genetics in Medicine"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0019|cit19","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1007\/8904_2013_250","article-title":"Successful desensitisation in a patient with CRIM-positive infantile-onset Pompe disease","volume":"12","author":"Baruteau","year":"2014","journal-title":"JIMD Reports"},{"issue":"11","key":"10.1002\/14651858.CD011539.pub2-BIB0020|cit20","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1038\/gim.2015.6","article-title":"CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy","volume":"17","author":"Berrier","year":"2015","journal-title":"Genetics in Medicine"},{"issue":"3","key":"10.1002\/14651858.CD011539.pub2-BIB0021|cit21","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1203\/01.pdr.0000233014.84318.4e","article-title":"Brain development in infantile-onset Pompe disease treated by enzyme replacement therapy","volume":"60","author":"Chien","year":"2006","journal-title":"Pediatric Research"},{"issue":"4","key":"10.1002\/14651858.CD011539.pub2-BIB0022|cit22","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/j.pedneo.2013.03.009","article-title":"Pompe disease: early diagnosis and early treatment make a difference","volume":"54","author":"Chien","year":"2013","journal-title":"Pediatrics and Neonatology"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0023|cit23","unstructured":"Eto Y The Japanese guideline for diagnosis and management of glycogen storage disease type II (Pompe disease) 3rd European Symposium on Steps Forward in Pompe Disease; 2009 Nov 20-21; Munich (Germany) 2010 S73 4"},{"issue":"4","key":"10.1002\/14651858.CD011539.pub2-BIB0024|cit24","doi-asserted-by":"crossref","first-page":"700","DOI":"10.1002\/ana.20807","article-title":"Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease","volume":"59","author":"Fukuda","year":"2006","journal-title":"Annals of Neurology"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0025|cit25","unstructured":"GRADE Working Group, McMaster University GRADEpro 2011"},{"issue":"4","key":"10.1002\/14651858.CD011539.pub2-BIB0026|cit26","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1016\/j.jclinepi.2010.04.026","article-title":"GRADE guidelines: 1. Introduction - GRADE evidence profiles and summary of findings tables","volume":"64","author":"Guyatt","year":"2011","journal-title":"Journal of Clinical Epidemiology"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0027|cit27","unstructured":"Higgins JPT Green S editor(s) Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 (updated March 2011). The Cochrane Collaboration, 2011 Available from handbook.cochrane.org"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0028|cit28","unstructured":"Hirschhorn R Reuser AJJ The metabolic and molecular bases of inherited disease. Glycogen storage disease type II: acid alpha-glucosidase (acid maltase) deficiency hommbid.mhmedical.com\/content.aspx?bookid=474&sectionid=45374135&Resultclick=2"},{"issue":"11","key":"10.1002\/14651858.CD011539.pub2-BIB0029|cit29","doi-asserted-by":"crossref","first-page":"e86821","DOI":"10.1172\/jci.insight.86821","article-title":"Durable and sustained immune tolerance to ERT in Pompe disease with entrenched immune responses","volume":"1","author":"Kazi","year":"2016","journal-title":"JCI Insight"},{"issue":"5","key":"10.1002\/14651858.CD011539.pub2-BIB0030|cit30","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1097\/01.gim.0000218152.87434.f3","article-title":"Pompe disease diagnosis and management guideline","volume":"8","author":"Kishnani","year":"2006","journal-title":"Genetics in Medicine"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0031|cit31","doi-asserted-by":"crossref","unstructured":"Kishnani PS Corzo D Leslie ND Gruskin D Van der Ploeg A Clancy JP Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease Pediatric Research 2009 329 35","DOI":"10.1203\/PDR.0b013e3181b24e94"},{"issue":"1","key":"10.1002\/14651858.CD011539.pub2-BIB0032|cit32","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1016\/j.nmd.2004.10.009","article-title":"Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial","volume":"15","author":"Klinge","year":"2005","journal-title":"Neuromuscular Disorders"},{"issue":"6","key":"10.1002\/14651858.CD011539.pub2-BIB0033|cit33","doi-asserted-by":"crossref","first-page":"E13","DOI":"10.1002\/humu.20745","article-title":"Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating","volume":"29","author":"Kroos","year":"2008","journal-title":"Human Mutation"},{"issue":"1","key":"10.1002\/14651858.CD011539.pub2-BIB0034|cit34","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1038\/gim.2011.4","article-title":"Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease","volume":"14","author":"Messinger","year":"2012","journal-title":"Genetics in Medicine"},{"issue":"S451","key":"10.1002\/14651858.CD011539.pub2-BIB0035|cit35","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1080\/08035320600618957","article-title":"Lysosomal dysfunction, cellular pathology and clinical symptoms: basic principles","volume":"95","author":"Reuser","year":"2006","journal-title":"Acta Paediatrica"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0036|cit36","unstructured":"Nordic Cochrane Centre, The Cochrane Collaboration Review Manager (RevMan) 2014"},{"issue":"2","key":"10.1002\/14651858.CD011539.pub2-BIB0037|cit37","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1067\/mpd.2000.107112","article-title":"Identification of two subtypes of infantile acid maltase deficiency","volume":"137","author":"Slonim","year":"2000","journal-title":"Journal of Pediatrics"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0038|cit38","unstructured":"Sterne J Egger M Moher D Chapter 10: Addressing reporting biases. In: Higgins JPT, Green S, editor(s). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1.0 (updated March 2011). The Cochrane Collaboration, 2011 Available from handbook.cochrane.org"},{"issue":"5","key":"10.1002\/14651858.CD011539.pub2-BIB0039|cit39","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1086\/522236","article-title":"Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance","volume":"81","author":"Sun","year":"2007","journal-title":"American Journal of Human Genetics"},{"issue":"2","key":"10.1002\/14651858.CD011539.pub2-BIB0040|cit40","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":"Gelder","year":"2015","journal-title":"Journal of Inherited Metabolic Disease"},{"issue":"9227","key":"10.1002\/14651858.CD011539.pub2-BIB0041|cit41","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1016\/S0140-6736(00)02533-2","article-title":"Recombinant human \u03b1-glucosidase from rabbit milk in Pompe patients","volume":"356","author":"Hout","year":"2000","journal-title":"Lancet"},{"issue":"2","key":"10.1002\/14651858.CD011539.pub2-BIB0042|cit42","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":"Hout","year":"2003","journal-title":"Pediatrics"},{"key":"10.1002\/14651858.CD011539.pub2-BIB0043|cit43","unstructured":"World Health Organization Bulletin of the World Health Organization: coming together to combat rare diseases www.who.int\/bulletin\/volumes\/90\/6\/12-020612\/en\/"},{"issue":"5","key":"10.1002\/14651858.CD011539.pub2-BIB0044|cit44","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1097\/01.gim.0000217781.66786.9b","article-title":"Comparison of maltose and acarbose as inhibitors of maltase-glucoamylase activity in assaying acid \u03b1-glucosidase activity in dried blood spots for the diagnosis of infantile Pompe disease","volume":"8","author":"Zhang","year":"2006","journal-title":"Genetics in Medicine"},{"issue":"3","key":"10.1002\/14651858.CD011539.pub2-BIB0045|cit45","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD011539","article-title":"Enzyme replacement therapy for infantile-onset Pompe disease","author":"Chen","year":"2015","journal-title":"Cochrane Database of Systematic Reviews"}],"container-title":["Cochrane Database of Systematic Reviews"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full\/10.1002\/14651858.CD011539.pub2","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,25]],"date-time":"2023-09-25T08:20:14Z","timestamp":1695630014000},"score":1,"resource":{"primary":{"URL":"http:\/\/doi.wiley.com\/10.1002\/14651858.CD011539.pub2"}},"subtitle":[],"editor":[{"name":"Cochrane Cystic Fibrosis and Genetic Disorders Group","sequence":"additional","affiliation":[],"role":[{"role":"editor","vocabulary":"crossref"}]}],"short-title":[],"issued":{"date-parts":[[2017,11,20]]},"references-count":45,"journal-issue":{"issue":"12","published-online":{"date-parts":[[2017,12]]}},"URL":"https:\/\/doi.org\/10.1002\/14651858.cd011539.pub2","relation":{},"ISSN":["1465-1858"],"issn-type":[{"value":"1465-1858","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017,11,20]]}}}