{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T07:37:45Z","timestamp":1792654665035,"version":"4.5.0"},"reference-count":17,"publisher":"Massachusetts Medical Society","issue":"20","license":[{"start":{"date-parts":[[2018,5,17]],"date-time":"2018-05-17T00:00:00Z","timestamp":1526515200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"DOI":"10.13039\/501100000272","name":"National Institute for Health Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000272","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011102","name":"European Union Seventh Framework Program Grand","doi-asserted-by":"publisher","award":["281234"],"award-info":[{"award-number":["281234"]}],"id":[{"id":"10.13039\/100011102","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2018,5,17]]},"DOI":"10.1056\/nejmoa1712649","type":"journal-article","created":{"date-parts":[[2018,4,24]],"date-time":"2018-04-24T16:15:13Z","timestamp":1524586513000},"page":"1898-1907","source":"Crossref","is-referenced-by-count":458,"title":["Study of Intraventricular Cerliponase Alfa for CLN2 Disease"],"prefix":"10.1056","volume":"378","author":[{"given":"Angela","family":"Schulz","sequence":"first","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Temitayo","family":"Ajayi","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Specchio","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emily","family":"de Los Reyes","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Gissen","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Douglas","family":"Ballon","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan P.","family":"Dyke","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Cahan","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Slasor","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Jacoby","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alfried","family":"Kohlsch\u00fctter","sequence":"additional","affiliation":[{"name":"From the Department of Pediatrics, University Medical Center Hamburg\u2013Eppendorf, Hamburg, Germany (A.S., A.K.); BioMarin Pharmaceutical, Novato, CA (T.A., H.C., P.S., D.J.); the Department of Neuroscience, Bambino Ges\u00f9 Children\u2019s Hospital, IRCCS, Rome (N.S.); Nationwide Children\u2019s Hospital and Ohio State University, Columbus (E.L.R.); UCL Great Ormond Street Institute of Child Health, London (P.G.); and the Citigroup Biomedical Imaging Center, Departments of Radiology and Genetic Medicine, Weill..."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"150","reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.277.5333.1802"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1086\/302427"},{"key":"e_1_3_5_4_2","volume-title":"The neuronal ceroid lipofuscinoses (Batten disease). 2nd ed.","author":"Mole SE","year":"2011","unstructured":"Mole SE, Williams RE, Goebe HH, eds. The neuronal ceroid lipofuscinoses (Batten disease). 2nd ed. Oxford, England: Oxford University Press, 2011."},{"key":"e_1_3_5_5_2","first-page":"682","article-title":"CLN2 disease (classic late infantile neuronal ceroid lipofuscinosis).","volume":"13","author":"Kohlsch\u00fctter A","year":"2016","unstructured":"Kohlsch\u00fctter A, Schulz A. CLN2 disease (classic late infantile neuronal ceroid lipofuscinosis). Pediatr Endocrinol Rev 2016;13:Suppl 1:682-688.27491216","journal-title":"Pediatr Endocrinol Rev"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbadis.2013.04.008"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.10660"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000267885.47092.40"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1055\/s-0036-1583730"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1090-3798(15)30049-0"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1750-3639.2004.tb00500.x"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2014.09.004"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2011.06.018"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1002\/jnr.23423"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.exer.2014.06.008"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.beem.2015.01.006"},{"key":"e_1_3_5_17_2","first-page":"S86","article-title":"Perspectives from B cell immunology: fact and fancy.","volume":"47","author":"Hunt SV","year":"2009","unstructured":"Hunt SV. Perspectives from B cell immunology: fact and fancy. Int J Clin Pharmacol Ther 2009;47:Suppl 1:S86-S99.20040318","journal-title":"Int J Clin Pharmacol Ther"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.11.001"}],"container-title":["New England Journal of Medicine"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nejm.org\/doi\/pdf\/10.1056\/NEJMoa1712649","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,3,27]],"date-time":"2024-03-27T00:46:43Z","timestamp":1711500403000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa1712649"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,5,17]]},"references-count":17,"journal-issue":{"issue":"20","published-print":{"date-parts":[[2018,5,17]]}},"alternative-id":["10.1056\/NEJMoa1712649"],"URL":"https:\/\/doi.org\/10.1056\/nejmoa1712649","relation":{},"ISSN":["0028-4793","1533-4406"],"issn-type":[{"value":"0028-4793","type":"print"},{"value":"1533-4406","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,5,17]]}}}