{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T08:48:43Z","timestamp":1790153323915,"version":"4.0.1"},"reference-count":26,"publisher":"Public Library of Science (PLoS)","issue":"6","license":[{"start":{"date-parts":[[2013,6,25]],"date-time":"2013-06-25T00:00:00Z","timestamp":1372118400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/publicdomain\/zero\/1.0\/"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0067052","type":"journal-article","created":{"date-parts":[[2013,6,25]],"date-time":"2013-06-25T21:08:19Z","timestamp":1372194499000},"page":"e67052","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":99,"title":["Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT"],"prefix":"10.1371","volume":"8","author":[{"given":"Suhrad G.","family":"Banugaria","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean N.","family":"Prater","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Trusha T.","family":"Patel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie M.","family":"DeArmey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christie","family":"Milleson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn B.","family":"Sheets","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deeksha S.","family":"Bali","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine W.","family":"Rehder","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julian A. J.","family":"Raiman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raymond A.","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francois","family":"Labarthe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Charrow","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Harmatz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pranesh","family":"Chakraborty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy S.","family":"Rosenberg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya S.","family":"Kishnani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2013,6,25]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1042\/bj0860011","article-title":"alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe&apos;s disease)","volume":"86","author":"HG Hers","year":"1963","journal-title":"Biochem J"},{"key":"ref2","unstructured":"Hirschhorn R, Reuser AJJ, editors (2009) Glycogen storage disease type II: acid a-glucosidase (acid maltase) deficiency New York: McGraw-Hill."},{"key":"ref3","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":"M Nicolino","year":"2009","journal-title":"Genet Med"},{"key":"ref4","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":"PS Kishnani","year":"2007","journal-title":"Neurology"},{"key":"ref5","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":"PS Kishnani","year":"2006","journal-title":"J Pediatr"},{"key":"ref6","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1203\/PDR.0b013e3181b24e94","article-title":"Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease","volume":"66","author":"PS Kishnani","year":"2009","journal-title":"Pediatr Res"},{"key":"ref7","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":"YH Chien","year":"2009","journal-title":"Pediatrics"},{"key":"ref8","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":"PS Kishnani","year":"2010","journal-title":"Mol Genet Metab"},{"key":"ref9","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":"SG Banugaria","year":"2011","journal-title":"Genet Med"},{"key":"ref10","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":"YH Messinger","year":"2012","journal-title":"Genet Med"},{"key":"ref11","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1056\/NEJMc0806809","article-title":"Elimination of antibodies to recombinant enzyme in Pompe&apos;s disease","volume":"360","author":"NJ Mendelsohn","year":"2009","journal-title":"N Engl J Med"},{"key":"ref12","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1016\/j.ymgme.2012.01.019","article-title":"Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells","volume":"105","author":"SG Banugaria","year":"2012","journal-title":"Mol Genet Metab"},{"key":"ref13","doi-asserted-by":"crossref","first-page":"e532","DOI":"10.1542\/peds.2003-0988-L","article-title":"Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease","volume":"114","author":"TE Hunley","year":"2004","journal-title":"Pediatrics"},{"key":"ref14","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1586\/eci.12.40","article-title":"Immune modulation in Pompe disease treated with enzyme replacement therapy","volume":"8","author":"SG Banugaria","year":"2012","journal-title":"Expert Rev Clin Immunol"},{"key":"ref15","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1002\/ajmg.c.31319","article-title":"Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience","volume":"160","author":"DS Bali","year":"2012","journal-title":"Am J Med Genet C Semin Med Genet"},{"key":"ref16","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1080\/01621459.1958.10501452","article-title":"Nonparametric Estimation from Incomplete Observations","volume":"53","author":"EL Kaplan","year":"1958","journal-title":"Journal of the American Statistical Association"},{"key":"ref17","doi-asserted-by":"crossref","first-page":"751","DOI":"10.1007\/s10545-010-9209-0","article-title":"CRIM-negative infantile Pompe disease: 42-month treatment outcome","volume":"33","author":"M Rohrbach","year":"2010","journal-title":"J Inherit Metab Dis"},{"key":"ref18","doi-asserted-by":"crossref","unstructured":"Khallaf HA, Propst J, Geffrard S, Botha E, Pervaiz MA (2013) CRIM-Negative Pompe Disease Patients with Satisfactory Clinical Outcomes on Enzyme Replacement Therapy. In: Zschocke J, Gibson KM, Brown G, Morava E, Peters V, editors. JIMD Reports - Case and Research Reports, 2012\/6: Springer Berlin Heidelberg. 133\u2013137.","DOI":"10.1016\/j.ymgme.2012.11.018"},{"key":"ref19","doi-asserted-by":"crossref","first-page":"583","DOI":"10.1016\/j.ymgme.2011.08.003","article-title":"Atypical immunologic response in a patient with CRIM-negative Pompe disease","volume":"104","author":"MA Abbott","year":"2011","journal-title":"Mol Genet Metab"},{"key":"ref20","doi-asserted-by":"crossref","unstructured":"Banugaria SG, Prater SN, McGann JK, Feldman JD, Tannenbaum JA, <etal>et al<\/etal>.. (2012) Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease. Genet Med.","DOI":"10.1038\/gim.2012.110"},{"key":"ref21","doi-asserted-by":"crossref","first-page":"564","DOI":"10.1002\/j.1875-9114.1993.tb02759.x","article-title":"Immunomodulation with intravenous immunoglobulin","volume":"13","author":"PD Hall","year":"1993","journal-title":"Pharmacotherapy"},{"key":"ref22","doi-asserted-by":"crossref","first-page":"448","DOI":"10.1016\/j.ymgme.2012.09.017","article-title":"Severely impaired health status at diagnosis of Pompe disease: A cross-sectional analysis to explore the potential utility of neonatal screening","volume":"107","author":"T Rigter","year":"2012","journal-title":"Mol Genet Metab"},{"key":"ref23","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1353\/hcr.0.0011","article-title":"Expanding newborn screening: process, policy, and priorities","volume":"38","author":"VA Moyer","year":"2008","journal-title":"Hastings Cent Rep"},{"key":"ref24","doi-asserted-by":"crossref","first-page":"110","DOI":"10.1038\/nature10134","article-title":"Intravenous gammaglobulin suppresses inflammation through a novel T(H)2 pathway","volume":"475","author":"RM Anthony","year":"2011","journal-title":"Nature"},{"key":"ref25","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1016\/j.beha.2011.02.009","article-title":"Rituximab resistance","volume":"24","author":"AR Rezvani","year":"2011","journal-title":"Best Pract Res Clin Haematol"},{"key":"ref26","doi-asserted-by":"crossref","first-page":"800","DOI":"10.1038\/gim.2012.44","article-title":"The emerging phenotype of long-term survivors with infantile Pompe disease","volume":"14","author":"SN Prater","year":"2012","journal-title":"Genet Med"}],"container-title":["PLoS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0067052","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,12]],"date-time":"2024-05-12T23:29:19Z","timestamp":1715556559000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0067052"}},"subtitle":[],"editor":[{"given":"Andrea","family":"Dardis","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2013,6,25]]},"references-count":26,"journal-issue":{"issue":"6","published-online":{"date-parts":[[2013,6,25]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0067052","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2013,6,25]]}}}