Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study.

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第一作者： Jerry,Vockley

第一单位： University of Pittsburgh, Pittsburgh, Pennsylvania, USA.

作者： Jerry,Vockley ^[1] ; Barbara,Burton ^[2] ; Gerard,Berry ^[3] ; Nicola,Longo ^[4] ; John,Phillips ^[5] ; Amarilis,Sanchez-Valle ^[6] ; Kimberly,Chapman ^[7] ; Pranoot,Tanpaiboon ^[7] ; Stephanie,Grunewald ^[8] ; Elaine,Murphy ^[9] ; Xiaoxiao,Lu ^[10] ; Jason,Cataldo ^[10]

作者单位： University of Pittsburgh, Pittsburgh, Pennsylvania, USA. ^[1] Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, USA. ^[2] Boston Children's Hospital, Boston, Massachusetts, USA. ^[3] University of Utah, Salt Lake City, Utah, USA. ^[4] Vanderbilt University Medical Center, Nashville, Tennessee, USA. ^[5] USF Health, Morsani College of Medicine, Tampa, Florida, USA. ^[6] Children's National Health System, Washington, District of Columbia, USA. ^[7] Great Ormond Street Hospital and Institute of Child Health, NIHR Biomedical Research Center (BRC), UCL, London, UK. ^[8] National Hospital for Neurology and Neurosurgery, London, UK. ^[9] Ultragenyx Pharmaceutical Inc., Novato, California, USA. ^[10]

关键词 cardiomyopathy long-chain fatty acid oxidation disorders (LC-FAOD)rhabdomyolysis triheptanoin (UX007)