{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,20]],"date-time":"2026-09-20T14:05:03Z","timestamp":1789913103796,"version":"4.0.1"},"reference-count":114,"publisher":"Informa UK Limited","issue":"5","license":[{"start":{"date-parts":[[2019,3,19]],"date-time":"2019-03-19T00:00:00Z","timestamp":1552953600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"Orphazyme A\/S"}],"content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Expert Opinion on Drug Discovery"],"published-print":{"date-parts":[[2019,5,4]]},"DOI":"10.1080\/17460441.2019.1588882","type":"journal-article","created":{"date-parts":[[2019,3,19]],"date-time":"2019-03-19T06:59:43Z","timestamp":1552978783000},"page":"499-509","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":37,"title":["Animal models for Niemann-Pick type C: implications for drug discovery & development"],"prefix":"10.1080","volume":"14","author":[{"given":"Cathrine K.","family":"Fog","sequence":"first","affiliation":[{"name":"Orphazyme A\/S, Copenhagen, Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Kirkegaard","sequence":"additional","affiliation":[{"name":"Orphazyme A\/S, Copenhagen, Denmark"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2019,3,19]]},"reference":[{"key":"CIT0001","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-16"},{"key":"CIT0002","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2015.25"},{"key":"CIT0003","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1212\/CPJ.0000000000000399","volume":"7","author":"Patterson MC","year":"2017","journal-title":"Neurol Clin Pract"},{"key":"CIT0004","doi-asserted-by":"publisher","DOI":"10.1111\/j.1600-0854.2010.01032.x"},{"key":"CIT0005","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-018-0880-9"},{"key":"CIT0006","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-018-0844-0"},{"key":"CIT0007","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M708735200"},{"key":"CIT0008","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M114.549915"},{"key":"CIT0009","first-page":"1","author":"Pipalia NH","year":"2017","journal-title":"J Lipid Res"},{"key":"CIT0010","doi-asserted-by":"publisher","DOI":"10.1007\/8904_2011_49"},{"key":"CIT0011","doi-asserted-by":"publisher","DOI":"10.1111\/febs.12954"},{"key":"CIT0012","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.06.004"},{"key":"CIT0013","first-page":"1","author":"Newton J","year":"2018","journal-title":"Adv Biol Regul"},{"key":"CIT0014","doi-asserted-by":"publisher","DOI":"10.1177\/0883073817741054"},{"key":"CIT0015","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-015-0274-1"},{"key":"CIT0016","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.M000257"},{"key":"CIT0017","doi-asserted-by":"publisher","DOI":"10.1002\/1873-3468.12196"},{"key":"CIT0018","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.M040618"},{"key":"CIT0019","first-page":"1","volume":"1","author":"Tortelli B","year":"2014","journal-title":"Hum Mol Genet"},{"key":"CIT0020","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3001417"},{"key":"CIT0021","doi-asserted-by":"publisher","DOI":"10.1016\/j.taap.2014.06.014"},{"key":"CIT0022","first-page":"1","author":"Cluzeau CVM","year":"2012","journal-title":"Hum Mol Genet"},{"key":"CIT0023","doi-asserted-by":"publisher","DOI":"10.1124\/jpet.116.232975"},{"key":"CIT0024","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.P000331"},{"key":"CIT0025","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aad9823"},{"key":"CIT0026","doi-asserted-by":"publisher","DOI":"10.1517\/17425251003601961"},{"key":"CIT0027","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-006-0363-3"},{"key":"CIT0028","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0047845"},{"key":"CIT0029","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318208f4ab"},{"key":"CIT0030","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3181e6210e"},{"key":"CIT0031","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2009.07.003"},{"key":"CIT0032","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.43.1_Part_1.61"},{"key":"CIT0033","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)31465-4"},{"key":"CIT0035","unstructured":"Orphazyme reports positive results from full data set of Phase II\/III arimoclomol trial in Niemann-Pick disease type C (NPC). Orphazyme. [Internet]. [cited 2019 Feb 19]. Available from: https:\/\/www.orphazyme.com\/news-feed\/2019\/1\/30\/orphazyme-reports-positive-results-from-full-data-set-of-phase-iiiii-arimoclomol-trial-in-niemann-pick-disease-type-c-npc"},{"key":"CIT0036","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.012385"},{"key":"CIT0037","doi-asserted-by":"publisher","DOI":"10.1016\/j.brainres.2016.03.007"},{"key":"CIT0038","doi-asserted-by":"publisher","DOI":"10.1007\/BF00334507"},{"key":"CIT0039","doi-asserted-by":"publisher","DOI":"10.1016\/S1096-7192(03)00074-X"},{"key":"CIT0040","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0067084"},{"key":"CIT0041","doi-asserted-by":"publisher","DOI":"10.1203\/PDR.0b013e318184d2ce"},{"key":"CIT0042","doi-asserted-by":"publisher","DOI":"10.1097\/NEN.0b013e31825414a6"},{"key":"CIT0043","doi-asserted-by":"publisher","DOI":"10.1097\/NEN.0b013e318286587f"},{"key":"CIT0044","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbalip.2004.08.011"},{"key":"CIT0045","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0112503"},{"key":"CIT0046","doi-asserted-by":"publisher","DOI":"10.1016\/0005-2760(80)90116-2"},{"key":"CIT0047","doi-asserted-by":"publisher","DOI":"10.1126\/science.277.5323.232"},{"key":"CIT0048","first-page":"140","volume":"108","author":"Morris MD","year":"1982","journal-title":"Am J Pathol"},{"key":"CIT0049","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr505"},{"key":"CIT0050","doi-asserted-by":"publisher","DOI":"10.1016\/S0166-4328(01)00380-1"},{"key":"CIT0051","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2012.01.020"},{"key":"CIT0052","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2009.07.010"},{"key":"CIT0053","doi-asserted-by":"publisher","DOI":"10.1016\/S0306-4522(01)00517-6"},{"key":"CIT0054","doi-asserted-by":"publisher","DOI":"10.1111\/j.1471-4159.1985.tb04044.x"},{"key":"CIT0055","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0178179"},{"key":"CIT0056","doi-asserted-by":"publisher","DOI":"10.1002\/cne.10522"},{"key":"CIT0057","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-015-7819-z"},{"key":"CIT0058","first-page":"1","author":"Walterfang M","year":"2012","journal-title":"AJNR Am J Neuroradiol"},{"key":"CIT0059","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2012.11.009"},{"key":"CIT0060","doi-asserted-by":"publisher","DOI":"10.3174\/ajnr.A2490"},{"key":"CIT0061","doi-asserted-by":"publisher","DOI":"10.1093\/jnen\/63.6.660"},{"key":"CIT0062","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.5981-10.2011"},{"key":"CIT0063","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1003462"},{"key":"CIT0064","author":"Vanier MT","year":"2014","journal-title":"J Inherit Metab Dis"},{"key":"CIT0065","doi-asserted-by":"publisher","DOI":"10.1016\/S0014-4886(03)00345-5"},{"key":"CIT0066","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0028777"},{"key":"CIT0067","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0048273"},{"key":"CIT0068","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.M700525-JLR200"},{"key":"CIT0069","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbrc.2011.08.096"},{"key":"CIT0070","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.4173-14.2015"},{"key":"CIT0071","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2018.05.001"},{"key":"CIT0072","doi-asserted-by":"publisher","DOI":"10.1093\/oxfordjournals.jhered.a109635"},{"key":"CIT0073","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0308456101"},{"key":"CIT0074","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0023677"},{"key":"CIT0075","doi-asserted-by":"publisher","DOI":"10.1002\/humu.10255"},{"key":"CIT0076","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1112751108"},{"key":"CIT0077","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M116.770578"},{"key":"CIT0078","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aad9407"},{"key":"CIT0079","doi-asserted-by":"publisher","DOI":"10.1002\/hep.22327"},{"key":"CIT0080","first-page":"1","author":"Fu R","year":"2013","journal-title":"Hum Mol Genet"},{"key":"CIT0081","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.M007211"},{"key":"CIT0082","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0012941"},{"key":"CIT0083","doi-asserted-by":"publisher","DOI":"10.1038\/srep41931"},{"key":"CIT0084","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/dds401"},{"key":"CIT0085","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.M012377"},{"key":"CIT0086","doi-asserted-by":"publisher","DOI":"10.1515\/hsz-2018-0118"},{"key":"CIT0087","doi-asserted-by":"publisher","DOI":"10.1155\/2011\/176802"},{"key":"CIT0088","doi-asserted-by":"publisher","DOI":"10.1242\/dev.02079"},{"key":"CIT0089","doi-asserted-by":"publisher","DOI":"10.1534\/genetics.105.046565"},{"key":"CIT0090","doi-asserted-by":"publisher","DOI":"10.1242\/dev.004572"},{"key":"CIT0091","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-9822(00)00468-1"},{"key":"CIT0092","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbagen.2007.06.004"},{"key":"CIT0093","doi-asserted-by":"publisher","DOI":"10.1038\/nrd.2017.214"},{"key":"CIT0094","first-page":"8:982","author":"Torres S","year":"2017","journal-title":"Front Physiol"},{"key":"CIT0095","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/dds090"},{"key":"CIT0096","doi-asserted-by":"publisher","DOI":"10.1016\/j.stemcr.2014.03.014"},{"key":"CIT0097","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-8-144"},{"key":"CIT0098","doi-asserted-by":"publisher","DOI":"10.18632\/oncotarget.19976"},{"key":"CIT0099","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-8-34"},{"key":"CIT0100","doi-asserted-by":"publisher","DOI":"10.3389\/fnins.2018.00646"},{"key":"CIT0101","doi-asserted-by":"publisher","DOI":"10.1016\/S0960-9822(01)00396-7"},{"key":"CIT0102","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0810895106"},{"key":"CIT0103","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.3010101"},{"key":"CIT0104","doi-asserted-by":"publisher","DOI":"10.1093\/jnen\/60.1.49"},{"key":"CIT0105","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M311591200"},{"key":"CIT0106","author":"Pentchev P","year":"1984","journal-title":"J Biol"},{"key":"CIT0107","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddw367"},{"key":"CIT0108","doi-asserted-by":"publisher","DOI":"10.1194\/jlr.M071274"},{"key":"CIT0109","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddy212"},{"key":"CIT0110","doi-asserted-by":"publisher","DOI":"10.1038\/nature08710"},{"key":"CIT0111","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2014.03.001"},{"key":"CIT0112","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.1014890108"},{"key":"CIT0113","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1510198"},{"key":"CIT0114","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2016.122"},{"key":"CIT0117","doi-asserted-by":"publisher","DOI":"10.1136\/jmedgenet-2016-104178"}],"container-title":["Expert Opinion on Drug Discovery"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/17460441.2019.1588882","content-type":"application\/pdf","content-version":"am","intended-application":"text-mining"},{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/17460441.2019.1588882","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,12,1]],"date-time":"2020-12-01T18:16:30Z","timestamp":1606846590000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.1080\/17460441.2019.1588882"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,3,19]]},"references-count":114,"journal-issue":{"issue":"5","published-online":{"date-parts":[[2019,2,27]]},"published-print":{"date-parts":[[2019,5,4]]}},"alternative-id":["10.1080\/17460441.2019.1588882"],"URL":"https:\/\/doi.org\/10.1080\/17460441.2019.1588882","relation":{},"ISSN":["1746-0441","1746-045X"],"issn-type":[{"value":"1746-0441","type":"print"},{"value":"1746-045X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,3,19]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=iedc20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=iedc20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2018-12-12","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-02-26","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-03-19","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}