{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T19:06:49Z","timestamp":1790276809184,"version":"4.1.0"},"reference-count":13,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2019,2,11]],"date-time":"2019-02-11T00:00:00Z","timestamp":1549843200000},"content-version":"vor","delay-in-days":41,"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\/501100003648","name":"National Medical Center","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003648","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008900","name":"University of South Florida","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008900","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013017","name":"Vanderbilt University Medical Center","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013017","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007747","name":"University of Utah","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007747","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"}]},{"DOI":"10.13039\/100013220","name":"Ultragenyx Pharmaceutical","doi-asserted-by":"publisher","award":["UX007\u2010CL201"],"award-info":[{"award-number":["UX007\u2010CL201"]}],"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":[[2019,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Long\u2010chain fatty acid oxidation disorders (LC\u2010FAOD) are rare disorders characterized by acute crises of energy metabolism and severe energy deficiency that may present with cardiomyopathy, hypoglycemia, and\/or rhabdomyolysis, which can lead to frequent hospitalizations and early death. An open\u2010label Phase 2 study evaluated the efficacy of UX007, an investigational odd\u2010carbon medium\u2010chain triglyceride, in 29 subjects with severe LC\u2010FAOD. UX007 was administered over 78\u2009weeks at a target dose of 25\u201335% total daily caloric intake (mean 27.5%). The frequency and duration of major clinical events (hospitalizations, emergency room visits, and emergency home interventions due to rhabdomyolysis, hypoglycemia, and cardiomyopathy) occurring during 78\u2009weeks of UX007 treatment was compared with the frequency and duration of events captured retrospectively from medical records for 78\u2009weeks before UX007 initiation. The mean annualized event rates decreased from 1.69 to 0.88 events\/year following UX007 initiation (\n                    <jats:italic>p<\/jats:italic>\n                    = 0.021; 48.1% reduction). The mean annualized duration rate decreased from 5.96 to 2.96\u2009days\/year (\n                    <jats:italic>p<\/jats:italic>\n                    = 0.028; 50.3% reduction). Hospitalizations due to rhabdomyolysis, the most common event, decreased from 1.03 to 0.63 events\/year (\n                    <jats:italic>p<\/jats:italic>\n                    = 0.104; 38.7% reduction). Initiation of UX007 eliminated hypoglycemia events leading to hospitalization (from 11 pre\u2010UX007 hospitalizations, 0.30 events\/year vs. 0;\n                    <jats:italic>p<\/jats:italic>\n                    = 0.067) and intensive care unit (ICU) care (from 2 pre\u2010UX007 ICU admissions, 0.05 events\/year vs. 0;\n                    <jats:italic>p<\/jats:italic>\n                    = 0.161) and reduced cardiomyopathy events (3 events vs. 1 event; 0.07 to 0.02 events\/year; 69.7% decrease). The majority of treatment\u2010related adverse events (AEs) were mild to moderate gastrointestinal symptoms, including diarrhea, vomiting, and abdominal or gastrointestinal pain, which can be managed with smaller, frequent doses mixed with food.\n                  <\/jats:p>","DOI":"10.1002\/jimd.12038","type":"journal-article","created":{"date-parts":[[2019,2,11]],"date-time":"2019-02-11T03:08:54Z","timestamp":1549854534000},"page":"169-177","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":56,"title":["Results from a 78\u2010week, single\u2010arm, open\u2010label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long\u2010chain fatty acid oxidation disorders (LC\u2010FAOD)"],"prefix":"10.1002","volume":"42","author":[{"given":"Jerry","family":"Vockley","sequence":"first","affiliation":[{"name":"School of Medicine and Graduate School of Public Health University of Pittsburgh  Pittsburgh Pennsylvania USA"},{"name":"Children\u2019s Hospital of Pittsburgh UPMC, 4401 Penn Avenue  Pittsburgh Pennsylvania 15224 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Burton","sequence":"additional","affiliation":[{"name":"Ann &amp; Robert H. Lurie Children's Hospital of Chicago  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerard T.","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":"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, UCL Institute of Child Health  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":"Alexandra","family":"Bowden","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wencong","family":"Chen","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chao\u2010Yin","family":"Chen","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"}]},{"given":"Deborah","family":"Marsden","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emil","family":"Kakkis","sequence":"additional","affiliation":[{"name":"Ultragenyx Pharmaceutical Inc.  Novato California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,2,11]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.10.0054712637"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-012-9542-6"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-009-1125-9"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-009-1126-8"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-006-0290-3"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/jcbfm.2012.151"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpendo.00384.2009"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI0215311151060"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000318283.42961.e92676979"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.06.0064561603"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2016.08.0085083220"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-017-0085-8"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2017.02.005"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fjimd.12038","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12038","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/jimd.12038","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/jimd.12038","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,10,20]],"date-time":"2023-10-20T02:08:41Z","timestamp":1697767721000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/jimd.12038"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,1]]},"references-count":13,"aliases":["10.1007\/s10545-018-0217-9"],"journal-issue":{"issue":"1","published-print":{"date-parts":[[2019,1]]}},"alternative-id":["10.1002\/jimd.12038"],"URL":"https:\/\/doi.org\/10.1002\/jimd.12038","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,1]]},"assertion":[{"value":"2019-02-11","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}