{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,14]],"date-time":"2026-05-14T23:23:57Z","timestamp":1778801037927,"version":"3.51.4"},"reference-count":18,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2019,12,29]],"date-time":"2019-12-29T00:00:00Z","timestamp":1577577600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["J Child Neurol"],"published-print":{"date-parts":[[2020,4]]},"abstract":"<jats:p>The objective of this review is to summarize the pharmacology, efficacy, and safety of cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Cerliponase alfa is recombinant human tripeptidyl peptidase 1 enzyme replacement therapy. A phase 1\/2 trial established the efficacy and safety of cerliponase alfa for treatment of neuronal ceroid lipofuscinosis type 2. Treatment with intracerebroventricular cerliponase alfa resulted in slower decline of motor and language functions compared with natural history controls. Common adverse events include convulsions, electrocardiography abnormalities, pyrexia, vomiting, and upper respiratory tract infections. Intracerebroventricular device\u2013related adverse events also occur. Cerliponase alfa is the first therapy for neuronal ceroid lipofuscinosis type 2 that targets the disease etiology. Cerliponase alfa is effective in delaying the progression of motor language decline for patients with neuronal ceroid lipofuscinosis type 2.<\/jats:p>","DOI":"10.1177\/0883073819895694","type":"journal-article","created":{"date-parts":[[2019,12,29]],"date-time":"2019-12-29T23:47:55Z","timestamp":1577663275000},"page":"348-353","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":26,"title":["Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2"],"prefix":"10.1177","volume":"35","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3386-154X","authenticated-orcid":false,"given":"Grace","family":"Lewis","sequence":"first","affiliation":[{"name":"Department Pharmacy Practice, MCPHS University School of Pharmacy\u2013Worcester\/Manchester, Worcester, MA, USA"},{"name":"Sanofi Genzyme, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amanda M.","family":"Morrill","sequence":"additional","affiliation":[{"name":"Department Pharmacy Practice, MCPHS University School of Pharmacy\u2013Worcester\/Manchester, Worcester, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephanie L.","family":"Conway-Allen","sequence":"additional","affiliation":[{"name":"Department Pharmacy Practice, MCPHS University School of Pharmacy\u2013Worcester\/Manchester, Worcester, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bernard","family":"Kim","sequence":"additional","affiliation":[{"name":"Department Pharmacy Practice, MCPHS University School of Pharmacy\u2013Worcester\/Manchester, Worcester, MA, USA"},{"name":"Sanofi Genzyme, Cambridge, MA, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2019,12,29]]},"reference":[{"key":"bibr1-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.07.011"},{"key":"bibr2-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-016-0414-2"},{"key":"bibr3-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30368-5"},{"key":"bibr4-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2017.01.034"},{"issue":"2","key":"bibr5-0883073819895694","first-page":"73","volume":"18","author":"Nita DA","year":"2016","journal-title":"Epileptic Disord"},{"key":"bibr6-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1016\/S0167-4838(98)00012-0"},{"key":"bibr7-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbadis.2012.08.012"},{"key":"bibr8-0883073819895694","volume-title":"Cork","author":"Brineura [summary of product characteristics]","year":"2018"},{"key":"bibr9-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1016\/j.clim.2018.09.003"},{"key":"bibr10-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1712649"},{"key":"bibr11-0883073819895694","doi-asserted-by":"publisher","DOI":"10.1177\/2326409818788382"},{"key":"bibr12-0883073819895694","unstructured":"Biomarin Pharmaceutical. A safety, tolerability, and efficacy study of intracerebroventricular BMN 190 in pediatric patients <18 years of age with CLN2 disease. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02678689. Published February 10, 2016. Accessed January 1, 2019."},{"key":"bibr13-0883073819895694","unstructured":"Biomarin Pharmaceutical. A multicenter, multi-national open-label program to provide BMN 190 to patients diagnosed with CLN2 disease. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02963350. Published November 15, 2016. Accessed January 1, 2019."},{"key":"bibr14-0883073819895694","unstructured":"Active ingredient: cerliponase alfa. RED BOOK Online. Micromedex Healthcare Series [database online]. Greenwood Village, CO: Truven Health Analytics. Accessed June 17, 2019."},{"key":"bibr15-0883073819895694","volume-title":"Published","author":"NICE","year":"2019"},{"key":"bibr16-0883073819895694","unstructured":"NICE. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 [ID943]. https:\/\/www.nice.org.uk\/guidance\/indevelopment\/gid-hst10008 . Published 2019. Accessed October 1, 2019."},{"key":"bibr17-0883073819895694","unstructured":"Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical, Inc; 2018."},{"key":"bibr18-0883073819895694","unstructured":"FDA Center for Drug Evaluation and Research. Risk assessment and risk mitigation review, application 761052. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2017\/761052Orig1s000TOC.cfm. 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