{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T20:26:19Z","timestamp":1792009579939,"version":"4.4.0"},"reference-count":23,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2020,9,14]],"date-time":"2020-09-14T00:00:00Z","timestamp":1600041600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013220","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013220","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2021,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Long\u2010chain fatty acid oxidation disorders (LC\u2010FAOD) are autosomal recessive conditions that impair conversion of long\u2010chain fatty acids into energy, leading to significant clinical symptoms. Triheptanoin is a highly purified, 7\u2010carbon chain triglyceride approved in the United States as a source of calories and fatty acids for treatment of pediatric and adult patients with molecularly confirmed LC\u2010FAOD. CL202 is an open\u2010label, long\u2010term extension study evaluating triheptanoin (Dojolvi) safety and efficacy in patients with LC\u2010FAOD. Patients rolled over from the CL201 triheptanoin clinical trial (rollover); were triheptanoin\u2010na\u00efve (na\u00efve); or had participated in investigator\u2010sponsored trials\/expanded access programs (IST\/other). Results focus on rollover and na\u00efve groups, as pretreatment data allow comparison. Primary outcomes were annual rate and duration of major clinical events (MCEs; rhabdomyolysis, hypoglycemia, and cardiomyopathy events). Seventy\u2010five patients were enrolled (24 rollover, 20 na\u00efve, 31 IST\/other). Mean study duration was 23.0 months for rollover, 15.7 months for na\u00efve, and 34.7 months for IST\/other. In the rollover group, mean annualized MCE rate decreased from 1.76 events\/year pre\u2010triheptanoin to 0.96 events\/year with triheptanoin (<jats:italic>P<\/jats:italic> =\u2009.0319). Median MCE duration was reduced by 66%. In the na\u00efve group, median annualized MCE rate decreased from 2.33 events\/year pre\u2010triheptanoin to 0.71 events\/year with triheptanoin (<jats:italic>P<\/jats:italic> =\u2009.1072). Median MCE duration was reduced by 80%. The most common related adverse events (AEs) were diarrhea, abdominal pain\/discomfort, and vomiting, most mild to moderate. Three patients had serious AEs (diverticulitis, ileus, rhabdomyolysis) possibly related to drug; all resolved. Two patients had AEs leading to death; neither drug related. Triheptanoin reduced rate and duration of MCEs. Safety was consistent with previous observations.<\/jats:p>","DOI":"10.1002\/jimd.12313","type":"journal-article","created":{"date-parts":[[2020,9,4]],"date-time":"2020-09-04T10:18:55Z","timestamp":1599214735000},"page":"253-263","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":59,"title":["Effects of triheptanoin (<scp>UX007<\/scp>) in patients with long\u2010chain fatty acid oxidation disorders: Results from an <scp>open\u2010label<\/scp>, <scp>long\u2010term<\/scp> extension study"],"prefix":"10.1002","volume":"44","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8180-6457","authenticated-orcid":false,"given":"Jerry","family":"Vockley","sequence":"first","affiliation":[{"name":"University of Pittsburgh  Pittsburgh Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Burton","sequence":"additional","affiliation":[{"name":"Ann &amp; Robert H. Lurie Children's Hospital  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerard","family":"Berry","sequence":"additional","affiliation":[{"name":"Boston Children's Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Longo","sequence":"additional","affiliation":[{"name":"University of Utah  Salt Lake City Utah USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Phillips","sequence":"additional","affiliation":[{"name":"Vanderbilt University Medical Center  Nashville Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amarilis","family":"Sanchez\u2010Valle","sequence":"additional","affiliation":[{"name":"USF Health Morsani College of Medicine  Tampa Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimberly","family":"Chapman","sequence":"additional","affiliation":[{"name":"Children's National Health System  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pranoot","family":"Tanpaiboon","sequence":"additional","affiliation":[{"name":"Children's National Health System  Washington District of Columbia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephanie","family":"Grunewald","sequence":"additional","affiliation":[{"name":"Great Ormond Street Hospital and Institute of Child Health NIHR Biomedical Research Center (BRC), UCL  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elaine","family":"Murphy","sequence":"additional","affiliation":[{"name":"National Hospital for Neurology and Neurosurgery  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoxiao","family":"Lu","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason","family":"Cataldo","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,9,14]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11154-018-9448-1"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.21037\/atm.2018.10.57"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.c.30087"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s10038-018-0527-7"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-016-0548-2"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.2008.148338"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2017.02.005"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9542-6"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-009-9001-1"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-009-1126-8"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.10.005"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpendo.00384.2009"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/jcbfm.2012.151"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-015-0236-7"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1007\/8904_2014_313"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/jimd.12038"},{"issue":"1","key":"e_1_2_10_18_1","article-title":"Development, validation, and norming of the SF\u201010 for Children Health Survey","volume":"15","author":"Baglama RN","year":"2006","journal-title":"Qual Life Res"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1186\/1477-7525-5-54"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-010-9090-x"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0008-6363(99)00263-1"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.06.006"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.08.008"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0131926"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12313","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/jimd.12313","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12313","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T06:25:23Z","timestamp":1692253523000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jimd.12313"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,9,14]]},"references-count":23,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2021,1]]}},"alternative-id":["10.1002\/jimd.12313"],"URL":"https:\/\/doi.org\/10.1002\/jimd.12313","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,9,14]]},"assertion":[{"value":"2020-01-27","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-09-01","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-09-14","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}