{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,2]],"date-time":"2026-09-02T22:25:46Z","timestamp":1788387946124,"version":"build-2803163510"},"reference-count":54,"publisher":"Informa UK Limited","license":[{"start":{"date-parts":[[2020,3,1]],"date-time":"2020-03-01T00:00:00Z","timestamp":1583020800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/3.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["TCRM"],"DOI":"10.2147\/tcrm.s241048","type":"journal-article","created":{"date-parts":[[2020,3,29]],"date-time":"2020-03-29T22:39:50Z","timestamp":1585521590000},"page":"213-222","source":"Crossref","is-referenced-by-count":48,"title":["<p>Changing Times for CLN2 Disease: The Era of Enzyme Replacement Therapy<\/p>"],"prefix":"10.2147","volume":"Volume 16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8120-0287","authenticated-orcid":true,"given":"Nicola","family":"Specchio","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2446-796X","authenticated-orcid":true,"given":"Nicola","family":"Pietrafusa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina","family":"Trivisano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2020,3]]},"reference":[{"key":"ref1","volume-title":"The Neuronal Ceroid Lipofuscinoses (Batten Disease)","year":"2011"},{"key":"ref2","doi-asserted-by":"publisher","first-page":"254","DOI":"10.1002\/ddrr.v17.3","volume":"17","author":"Bennett","year":"2013","journal-title":"Dev Disabil Res Rev"},{"key":"ref3","doi-asserted-by":"publisher","first-page":"102","DOI":"10.1016\/j.pediatrneurol.2017.01.034","volume":"69","author":"Williams","year":"2017","journal-title":"Pediatr Neurol"},{"key":"ref4","doi-asserted-by":"publisher","first-page":"183","DOI":"10.1212\/WNL.0b013e31825f0547","volume":"79","author":"Williams","year":"2012","journal-title":"Neurology"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"661","DOI":"10.1007\/s00415-003-1050-z","volume":"250","author":"Teixeira","year":"2003","journal-title":"J Neurol"},{"key":"ref6","doi-asserted-by":"publisher","first-page":"536","DOI":"10.1002\/(ISSN)1096-8628","volume":"42","author":"Claussen","year":"1992","journal-title":"Am J Med Genet"},{"key":"ref7","doi-asserted-by":"publisher","first-page":"213","DOI":"10.1111\/j.1399-0004.2008.01054.x","volume":"74","author":"Moore","year":"2008","journal-title":"Clin Genet"},{"key":"ref8","doi-asserted-by":"publisher","first-page":"7135","DOI":"10.1074\/jbc.M211872200","volume":"278","author":"Golabek","year":"2003","journal-title":"J Biol Chem"},{"key":"ref9","doi-asserted-by":"publisher","first-page":"8","DOI":"10.1186\/1472-6807-3-8","volume":"3","author":"Wlodawer","year":"2003","journal-title":"BMC Struct Biol"},{"key":"ref10","doi-asserted-by":"publisher","first-page":"1795","DOI":"10.1016\/j.bbadis.2012.08.012","volume":"1832","author":"Haltia","year":"2013","journal-title":"Biochim Biophys Acta"},{"key":"ref11","doi-asserted-by":"publisher","first-page":"147","DOI":"10.1242\/dmm.018820","volume":"8","author":"Phillips","year":"2015","journal-title":"Dis Model Mech"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1016\/j.jmb.2020.02.012","author":"Zatyka","year":"2020","journal-title":"J Mol Biol"},{"key":"ref13","doi-asserted-by":"publisher","first-page":"897","DOI":"10.1242\/dmm.029280","volume":"10","author":"Stumpf","year":"2017","journal-title":"Dis Model Mech"},{"key":"ref14","doi-asserted-by":"publisher","first-page":"469","DOI":"10.3390\/cells8050469","volume":"8","author":"Smith","year":"2019","journal-title":"Cells"},{"key":"ref15","doi-asserted-by":"publisher","first-page":"1488","DOI":"10.1093\/brain\/awt043","volume":"136","author":"Mahmood","year":"2013","journal-title":"Brain"},{"key":"ref16","doi-asserted-by":"publisher","first-page":"9117","DOI":"10.1523\/JNEUROSCI.2729-04.2004","volume":"24","author":"Sleat","year":"2004","journal-title":"J Neurosci"},{"key":"ref17","doi-asserted-by":"publisher","first-page":"e0176526","DOI":"10.1371\/journal.pone.0176526","volume":"12","author":"Geraets","year":"2017","journal-title":"PLoS One"},{"key":"ref18","doi-asserted-by":"publisher","first-page":"254","DOI":"10.1016\/j.ymgme.2006.02.016","volume":"89","author":"Awano","year":"2006","journal-title":"Mol Genet Metab"},{"key":"ref19","doi-asserted-by":"publisher","first-page":"1924","DOI":"10.1002\/humu.v40.11","volume":"40","author":"Gardner","year":"2019","journal-title":"Hum Mutat"},{"key":"ref20","doi-asserted-by":"publisher","first-page":"582","DOI":"10.1016\/S2352-4642(18)30179-2","volume":"2","author":"Nickel","year":"2018","journal-title":"Lancet Child Adolesc Health"},{"key":"ref21","first-page":"682","volume":"13","author":"Kohlsch\u00fctter","year":"2016","journal-title":"Pediatr Endocrinol Rev"},{"key":"ref22","doi-asserted-by":"publisher","first-page":"315","DOI":"10.1007\/s40263-019-00620-8","volume":"33","author":"Kohlsch\u00fctter","year":"2019","journal-title":"CNS Drugs"},{"key":"ref23","doi-asserted-by":"publisher","first-page":"521","DOI":"10.1212\/01.wnl.0000267885.47092.40","volume":"69","author":"Worgall","year":"2007","journal-title":"Neurology"},{"key":"ref24","doi-asserted-by":"publisher","first-page":"1801","DOI":"10.1016\/j.bbadis.2013.04.008","volume":"1832","author":"Schulz","year":"2013","journal-title":"Biochim Biophys Acta"},{"key":"ref25","doi-asserted-by":"publisher","first-page":"347","DOI":"10.1002\/(ISSN)1096-8628","volume":"112","author":"Steinfeld","year":"2002","journal-title":"Am J Med Genet"},{"key":"ref26","doi-asserted-by":"publisher","first-page":"470","DOI":"10.1177\/0883073812448459","volume":"28","author":"P\u00e9rez-Poyato","year":"2013","journal-title":"J Child Neurol"},{"key":"ref27","doi-asserted-by":"publisher","first-page":"e73128","DOI":"10.1371\/journal.pone.0073128","volume":"8","author":"Orlin","year":"2013","journal-title":"PLoS One"},{"key":"ref28","doi-asserted-by":"publisher","first-page":"215","DOI":"10.5935\/0004-2749.20170053","volume":"80","author":"Quagliato","year":"2017","journal-title":"Arq Bras Oftalmol"},{"key":"ref29","doi-asserted-by":"publisher","first-page":"160","DOI":"10.1016\/j.ymgme.2016.07.011","volume":"119","author":"Fietz","year":"2016","journal-title":"Mol Genet Metab"},{"key":"ref30","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1177\/2326409818788382","volume":"6","author":"Wyrwich","year":"2018","journal-title":"J Inborn Errors Metab Screen"},{"key":"ref31","doi-asserted-by":"publisher","first-page":"1380","DOI":"10.1111\/epi.2017.58.issue-8","volume":"58","author":"Specchio","year":"2017","journal-title":"Epilepsia"},{"key":"ref32","doi-asserted-by":"publisher","first-page":"1938","DOI":"10.3174\/ajnr.A4816","volume":"37","author":"L\u00f6bel","year":"2016","journal-title":"AJNR Am J Neuroradiol"},{"key":"ref33","doi-asserted-by":"publisher","first-page":"1160","DOI":"10.3174\/ajnr.A4669","volume":"37","author":"Dyke","year":"2016","journal-title":"AJNR Am J Neuroradiol"},{"key":"ref34","doi-asserted-by":"publisher","first-page":"1475","DOI":"10.1177\/0883073816658659","volume":"31","author":"Albert","year":"2016","journal-title":"J Child Neurol"},{"key":"ref35","doi-asserted-by":"publisher","first-page":"85","DOI":"10.1016\/j.pediatrneurol.2013.08.008","volume":"50","author":"Jadav","year":"2014","journal-title":"Pediatr Neurol"},{"key":"ref36","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1111\/dmcn.14346","volume":"62","author":"Dozi\u00e8res-Puyravel","year":"2019","journal-title":"Dev Med Child Neurol"},{"key":"ref37","unstructured":"ClinicalTrials.gov. Trials in patients with CLN2 disease. Available from: https:\/\/clinicaltrials.gov\/ct2\/results?cond=CLN2+disease&term=&cntry=&state=&city=&dist=. Accessed March 3, 2020."},{"key":"ref38","doi-asserted-by":"publisher","first-page":"313ra180","DOI":"10.1126\/scitranslmed.aac6191","volume":"7","author":"Katz","year":"2015","journal-title":"Sci Transl Med"},{"key":"ref39","doi-asserted-by":"publisher","first-page":"1334","DOI":"10.1523\/JNEUROSCI.2676-05.2006","volume":"26","author":"Passini","year":"2006","journal-title":"J Neurosci"},{"key":"ref40","doi-asserted-by":"publisher","first-page":"463","DOI":"10.1089\/hum.2008.022","volume":"19","author":"Worgall","year":"2008","journal-title":"Hum Gene Ther"},{"key":"ref41","doi-asserted-by":"publisher","first-page":"643","DOI":"10.3171\/2013.3.PEDS12397","volume":"11","author":"Selden","year":"2013","journal-title":"J Neurosurg Pediatr"},{"key":"ref42","doi-asserted-by":"publisher","first-page":"2005","DOI":"10.1093\/hmg\/ddt596","volume":"23","author":"Lojewski","year":"2014","journal-title":"Hum Mol Genet"},{"key":"ref43","doi-asserted-by":"publisher","first-page":"1247","DOI":"10.1007\/s40265-017-0771-8","volume":"77","author":"Markham","year":"2017","journal-title":"Drugs"},{"key":"ref44","unstructured":"Cerliponase Alfa (Brineura) [Summary of Product Characteristics]. Cork, Ireland: BioMarin International Limited; 2017. Available from: https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/brineura-epar-product-information_en.pdf. Accessed March 23, 2020."},{"key":"ref45","unstructured":"Cerliponase Alfa (Brineura) [Prescribing Information]. Novato, CA: BioMarin Pharmaceutical Inc; 2017. Available from: https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2017\/761052lbl.pdf. Accessed March 23, 2020."},{"key":"ref46","doi-asserted-by":"publisher","first-page":"798","DOI":"10.1111\/j.1601-183X.2011.00718.x","volume":"10","author":"Sanders","year":"2011","journal-title":"Genes Brain Behav"},{"key":"ref47","doi-asserted-by":"publisher","first-page":"2686","DOI":"10.1167\/iovs.08-1712","volume":"49","author":"Katz","year":"2008","journal-title":"Invest Ophthalmol Vis Sci"},{"key":"ref48","doi-asserted-by":"publisher","first-page":"649","DOI":"10.1038\/mt.2008.9","volume":"16","author":"Chang","year":"2008","journal-title":"Mol Ther"},{"key":"ref49","doi-asserted-by":"publisher","first-page":"325","DOI":"10.1016\/j.ymgme.2011.06.018","volume":"104","author":"Vuillemenot","year":"2011","journal-title":"Mol Genet Metab"},{"key":"ref50","doi-asserted-by":"publisher","first-page":"281","DOI":"10.1016\/j.ymgme.2014.09.004","volume":"114","author":"Vuillemenot","year":"2015","journal-title":"Mol Genet Metab"},{"key":"ref51","doi-asserted-by":"publisher","first-page":"1591","DOI":"10.1002\/jnr.23423","volume":"92","author":"Katz","year":"2014","journal-title":"J Neurosci Res"},{"key":"ref52","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1016\/j.taap.2014.03.005","volume":"277","author":"Vuillemenot","year":"2014","journal-title":"Toxicol Appl Pharmacol"},{"key":"ref53","doi-asserted-by":"publisher","first-page":"1898","DOI":"10.1056\/NEJMoa1712649","volume":"378","author":"Schulz","year":"2018","journal-title":"N Engl J Med"},{"key":"ref54","year":"2019"}],"container-title":["Therapeutics and Clinical Risk Management"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.dovepress.com\/getfile.php?fileID=57092","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.dovepress.com\/getfile.php?fileID=57092","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,3,29]],"date-time":"2020-03-29T22:39:55Z","timestamp":1585521595000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.dovepress.com\/changing-times-for-cln2-disease-the-era-of-enzyme-replacement-therapy-peer-reviewed-article-TCRM"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,3]]},"references-count":54,"URL":"https:\/\/doi.org\/10.2147\/tcrm.s241048","relation":{},"ISSN":["1178-203X"],"issn-type":[{"value":"1178-203X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,3]]}}}