Page last updated: 2024-08-25

triheptanoin and Lipid Metabolism, Inborn Error

triheptanoin has been researched along with Lipid Metabolism, Inborn Error in 19 studies

Research

Studies (19)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's0 (0.00)29.6817
2010's7 (36.84)24.3611
2020's12 (63.16)2.80

Authors

AuthorsStudies
Bedrosian, CL; Enns, GM; Lu, X; Marsden, D; Rahman, S; Ramirez, AN; Ray, K; Reineking, B; Vockley, J1
Gosselin, NH; Jomphe, C; Lee, SK; McKeever, K; Putnam, W1
Gokmen-Ozel, H; Kadayifcilar, S; Kahraman, AB; Sivri, S; Yildiz, Y1
Berry, G; Burton, BK; Chapman, KA; Grunewald, S; Longo, N; Lu, X; Murphy, E; Phillips, J; Pisani, L; Rahman, S; Ramirez, AN; Ray, K; Reineking, B; Sanchez-Valle, A; Tanpaiboon, P; Vockley, J1
Berry, G; Burton, B; Cataldo, J; Chapman, K; Grunewald, S; Longo, N; Lu, X; Murphy, E; Phillips, J; Sanchez-Valle, A; Tanpaiboon, P; Vockley, J1
Shirley, M1
Chang, IJ; Gunnarson, M; Hahn, S; Lam, C; McKean, KN; Merritt, JL; Norris, MK; Scott, AI; Sullivan, S; Sun, A; Thies, JM1
Hufgard-Leitner, M; Jörg-Streller, M; Karall, D; Konstantopoulou, V; Scholl-Bürgi, S; Spenger, J; Stock, K; Zöggeler, T1
Barshop, BA; Chan, B; Gangoiti, JA; Gaston, G; Gillingham, MB; Harding, CO; Winn, S1
Cataldo, J; Chen, CY; Dwyer, L; Longo, N; Madden, M; Mu, Y; Vockley, J1
Akinshola, E; Beitz, J; Chakder, S; Doan, J; Donohue, K; Johnson, LL; Ma, L; Meyer, J; Pacanowski, M; Wang, J; Yi, S; Zand, D1
Gupta, M; Lee, SK; McKeever, K; Shi, J1
Burchill, LJ; DeLany, JP; Deward, S; El-Gharbawy, AH; Gillingham, MB; Goldstein, A; Goodpaster, B; Harding, CO; Heitner, SB; Lasarev, MR; Martin, J; Matern, D; Pollaro, J; Rose, S; Shoemaker, J; Vockley, J1
Shoffner, JM1
Behringer, S; Spiekerkoetter, U; Tucci, S1
Brunengraber, H; Roe, CR1
Abdenur, JE; Charrow, J; Conway, R; Diaz, GA; Enns, GM; Eswara, M; Ganesh, J; Marsden, DL; McCracken, E; Sanchez-de-Toledo, J; Starr, J; Vockley, J; Wang, R1
Beermann, F; Behringer, S; Floegel, U; Spiekerkoetter, U; Tucci, S1
Berry, GT; Bowden, A; Burton, B; Cataldo, J; Grunewald, S; Humphrey, R; Kakkis, E; Longo, N; Marsden, DL; Mayhew, J; Murphy, E; Phillips, J; Sanchez-Valle, A; Tanpaiboon, P; Vockley, J; Zhang, L1

Reviews

1 review(s) available for triheptanoin and Lipid Metabolism, Inborn Error

ArticleYear
Triheptanoin: First Approval.
    Drugs, 2020, Volume: 80, Issue:15

    Topics: Animals; Clinical Trials, Phase II as Topic; Clinical Trials, Phase III as Topic; Disease Models, Animal; Drug Approval; Drug Evaluation, Preclinical; Energy Metabolism; Fatty Acids; Humans; Lipid Metabolism, Inborn Errors; Oxidation-Reduction; Treatment Outcome; Triglycerides; United States; United States Food and Drug Administration

2020

Trials

7 trial(s) available for triheptanoin and Lipid Metabolism, Inborn Error

ArticleYear
Triheptanoin for the treatment of long-chain fatty acid oxidation disorders: Final results of an open-label, long-term extension study.
    Journal of inherited metabolic disease, 2023, Volume: 46, Issue:5

    Topics: Adult; Child; Fatty Acids; Humans; Lipid Metabolism, Inborn Errors; Oxidation-Reduction; Triglycerides

2023
Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study.
    Journal of inherited metabolic disease, 2021, Volume: 44, Issue:1

    Topics: Adolescent; Adult; Cardiomyopathies; Child; Child, Preschool; Fatty Acids; Female; Humans; Hypoglycemia; Infant; Lipid Metabolism, Inborn Errors; Male; Middle Aged; Oxidation-Reduction; Rhabdomyolysis; Triglycerides; United Kingdom; United States; Young Adult

2021
Dietary management and major clinical events in patients with long-chain fatty acid oxidation disorders enrolled in a phase 2 triheptanoin study.
    Clinical nutrition ESPEN, 2021, Volume: 41

    Topics: Fatty Acids; Humans; Lipid Metabolism, Inborn Errors; Retrospective Studies; Triglycerides

2021
Regulatory news: Dojolvi (triheptanoin) as a source of calories and fatty acids in long-chain fatty acid oxidation disorders: FDA approval summary.
    Journal of inherited metabolic disease, 2021, Volume: 44, Issue:3

    Topics: Adolescent; Adult; Child; Double-Blind Method; Drug Approval; Fatty Acids; Female; Humans; Lipid Metabolism, Inborn Errors; Male; Middle Aged; Oxidation-Reduction; Triglycerides; United States; United States Food and Drug Administration; Young Adult

2021
The Pharmacokinetics of Triheptanoin and Its Metabolites in Healthy Subjects and Patients With Long-Chain Fatty Acid Oxidation Disorders.
    Clinical pharmacology in drug development, 2021, Volume: 10, Issue:11

    Topics: 3-Hydroxybutyric Acid; Adolescent; Adult; Child; Cross-Over Studies; Fatty Acids; Female; Healthy Volunteers; Heptanoates; Humans; Lipid Metabolism, Inborn Errors; Male; Middle Aged; Oxidation-Reduction; Triglycerides; Young Adult

2021
Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial.
    Journal of inherited metabolic disease, 2017, Volume: 40, Issue:6

    Topics: Acyl-CoA Dehydrogenase, Long-Chain; Adolescent; Adult; Caprylates; Cardiomyopathies; Carnitine; Child; Dietary Fats; Double-Blind Method; Exercise; Fatty Acids; Female; Humans; Lipid Metabolism, Inborn Errors; Male; Middle Aged; Mitochondrial Myopathies; Mitochondrial Trifunctional Protein; Nervous System Diseases; Oxidation-Reduction; Rhabdomyolysis; Triglycerides; Young Adult

2017
UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24weeks of treatment.
    Molecular genetics and metabolism, 2017, Volume: 120, Issue:4

    Topics: Adolescent; Adult; Child; Child, Preschool; Fatty Acids; Female; Humans; Infant; Infant, Newborn; Lipid Metabolism, Inborn Errors; Male; Middle Aged; Physical Endurance; Prospective Studies; Quality of Life; Treatment Outcome; Triglycerides; Walk Test; Young Adult

2017

Other Studies

11 other study(ies) available for triheptanoin and Lipid Metabolism, Inborn Error

ArticleYear
Response to triheptanoin therapy in critically ill patients with LC-FAOD: Report of patients treated through an expanded access program.
    Molecular genetics and metabolism, 2022, Volume: 136, Issue:2

    Topics: Cardiomyopathies; Critical Illness; Fatty Acids; Humans; Infant; Lipid Metabolism, Inborn Errors; Oxidation-Reduction; Retrospective Studies; Stroke Volume; Triglycerides; Ventricular Function, Left

2022
Population Pharmacokinetics of Heptanoate in Healthy Subjects and Patients With Long-Chain Fatty Acid Oxidation Disorders Treated With Triheptanoin.
    Clinical pharmacology in drug development, 2022, Volume: 11, Issue:11

    Topics: Adult; Child; Fatty Acids; Healthy Volunteers; Heptanoates; Humans; Lipid Metabolism, Inborn Errors; Triglycerides

2022
Successful management of rhabdomyolysis with triheptanoin in a child with severe long-chain 3-hydroxyacyl-coenzyme A dehydrogenase (LCHAD) deficiency.
    Neuromuscular disorders : NMD, 2023, Volume: 33, Issue:4

    Topics: 3-Hydroxyacyl CoA Dehydrogenases; Child; Coenzyme A; Female; Humans; Infant; Lipid Metabolism, Inborn Errors; Oxidation-Reduction; Rhabdomyolysis; Triglycerides

2023
Tutorial: Triheptanoin and Nutrition Management for Treatment of Long-Chain Fatty Acid Oxidation Disorders.
    JPEN. Journal of parenteral and enteral nutrition, 2021, Volume: 45, Issue:2

    Topics: Carnitine; Fatty Acids; Humans; Lipid Metabolism, Inborn Errors; Oxidation-Reduction; Triglycerides

2021
Long-term experience with triheptanoin in 12 Austrian patients with long-chain fatty acid oxidation disorders.
    Orphanet journal of rare diseases, 2021, 01-14, Volume: 16, Issue:1

    Topics: Austria; Child; Fatty Acids; Humans; Lipid Metabolism, Inborn Errors; Prospective Studies; Retrospective Studies; Triglycerides

2021
Cardiac tissue citric acid cycle intermediates in exercised very long-chain acyl-CoA dehydrogenase-deficient mice fed triheptanoin or medium-chain triglyceride.
    Journal of inherited metabolic disease, 2020, Volume: 43, Issue:6

    Topics: Acyl-CoA Dehydrogenase, Long-Chain; Animals; Citric Acid Cycle; Congenital Bone Marrow Failure Syndromes; Dietary Fats; Fatty Acids; Female; Lipid Metabolism, Inborn Errors; Liver; Male; Mice; Mitochondrial Diseases; Muscular Diseases; Myocardium; Oxidation-Reduction; Triglycerides

2020
Concerning "Triheptanoin vs trioctanoin for long-chain fatty acid oxidation disorders: A double blinded, randomized controlled trial" by Gillingham et al.
    Journal of inherited metabolic disease, 2019, Volume: 42, Issue:3

    Topics: Caprylates; Fatty Acids; Humans; Lipid Metabolism, Inborn Errors; Oxidation-Reduction; Triglycerides

2019
De novo fatty acid biosynthesis and elongation in very long-chain acyl-CoA dehydrogenase-deficient mice supplemented with odd or even medium-chain fatty acids.
    The FEBS journal, 2015, Volume: 282, Issue:21

    Topics: Acyl-CoA Dehydrogenase, Long-Chain; Animals; Congenital Bone Marrow Failure Syndromes; Dietary Fats; Disease Models, Animal; Fatty Acids; Female; Lipid Metabolism, Inborn Errors; Liver; Male; Mice; Mice, 129 Strain; Mice, Inbred C57BL; Mice, Knockout; Mitochondrial Diseases; Muscular Diseases; Myocardium; Oxidation-Reduction; Peroxisomes; Triglycerides

2015
Anaplerotic treatment of long-chain fat oxidation disorders with triheptanoin: Review of 15 years Experience.
    Molecular genetics and metabolism, 2015, Volume: 116, Issue:4

    Topics: Administration, Oral; Adolescent; Adult; Carnitine; Child; Child, Preschool; Citric Acid; Fatty Acids; Female; Humans; Infant; Infant, Newborn; Lactic Acid; Lipid Metabolism, Inborn Errors; Malates; Male; Middle Aged; Mitochondrial Diseases; Oxidation-Reduction; Succinic Acid; Survival Analysis; Treatment Outcome; Triglycerides

2015
Triheptanoin treatment in patients with pediatric cardiomyopathy associated with long chain-fatty acid oxidation disorders.
    Molecular genetics and metabolism, 2016, Volume: 119, Issue:3

    Topics: Adolescent; Cardiomyopathies; Child; Child, Preschool; Clinical Trials as Topic; Energy Metabolism; Fatty Acids; Female; Humans; Infant; Infant, Newborn; Lipid Metabolism, Inborn Errors; Male; Oxidation-Reduction; Triglycerides

2016
Triheptanoin: long-term effects in the very long-chain acyl-CoA dehydrogenase-deficient mouse.
    Journal of lipid research, 2017, Volume: 58, Issue:1

    Topics: Acyl-CoA Dehydrogenase, Long-Chain; Animals; Cardiomyopathies; Energy Metabolism; Fatty Acids; Heptanoic Acids; Humans; Lipid Metabolism, Inborn Errors; Liver; Mice; Oxidation-Reduction; Oxidative Stress; Triglycerides

2017