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Triheptanoin

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https://www.readbyqxmd.com/read/29110179/a-double-blind-placebo-controlled-trial-of-triheptanoin-in-adult-polyglucosan-body-disease-and-open-label-long-term-outcome
#1
Raphael Schiffmann, Mary E Wallace, Daisy Rinaldi, Isabelle Ledoux, Marie-Pierre Luton, Scott Coleman, H Orhan Akman, Karine Martin, Jean-Yves Hogrel, Derek Blankenship, Jacob Turner, Fanny Mochel
BACKGROUND: Adult polyglucosan body disease (APBD) is a progressive neurometabolic disorder caused by a deficiency of glycogen branching enzyme. We tested the efficacy of triheptanoin as a therapy for patients with APBD based on the hypothesis that decreased glycogen degradation leads to brain energy deficit. METHODS AND RESULTS: This was a two-site, randomized crossover trial of 23 patients (age 35-73 years; 63% men) who received triheptanoin or vegetable oil as placebo...
November 6, 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28969699/a-randomized-controlled-double-blind-crossover-trial-of-triheptanoin-in-alternating-hemiplegia-of-childhood
#2
Elodie Hainque, Samantha Caillet, Sandrine Leroy, Constance Flamand-Roze, Isaac Adanyeguh, Fanny Charbonnier-Beaupel, Maryvonne Retail, Benjamin Le Toullec, Mariana Atencio, Sophie Rivaud-Péchoux, Vanessa Brochard, Florence Habarou, Chris Ottolenghi, Florence Cormier, Aurélie Méneret, Marta Ruiz, Mohamed Doulazmi, Anne Roubergue, Jean-Christophe Corvol, Marie Vidailhet, Fanny Mochel, Emmanuel Roze
BACKGROUND: Based on the hypothesis of a brain energy deficit, we investigated the safety and efficacy of triheptanoin on paroxysmal episodes in patients with alternating hemiplegia of childhood due to ATP1A3 mutations. METHODS: We conducted a randomized, double-blind, placebo-controlled crossover study of triheptanoin, at a target dose corresponding to 30% of daily calorie intake, in ten patients with alternating hemiplegia of childhood due to ATP1A3 mutations...
October 2, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28932990/treatment-opportunities-in-patients-with-metabolic-myopathies
#3
REVIEW
Mette Cathrine Ørngreen, John Vissing
Metabolic myopathies are disorders affecting utilization of carbohydrates or fat in the skeletal muscle. Adult patients with metabolic myopathies typically present with exercise-induced pain, contractures or stiffness, fatigue, and myoglobinuria. Symptoms are related to energy failure. Purpose of review In this review, the current treatment options, including exercise therapy, dietary treatment, pharmacological supplementation, gene transcription, and enzyme replacement therapy, are described. Recent findings Recognition of the metabolic block in the metabolic myopathies has started the development of new therapeutic options...
September 21, 2017: Current Treatment Options in Neurology
https://www.readbyqxmd.com/read/28871440/triheptanoin-versus-trioctanoin-for-long-chain-fatty-acid-oxidation-disorders-a-double-blinded-randomized-controlled-trial
#4
Melanie B Gillingham, Stephen B Heitner, Julie Martin, Sarah Rose, Amy Goldstein, Areeg Hassan El-Gharbawy, Stephanie Deward, Michael R Lasarev, Jim Pollaro, James P DeLany, Luke J Burchill, Bret Goodpaster, James Shoemaker, Dietrich Matern, Cary O Harding, Jerry Vockley
BACKGROUND: Observational reports suggest that supplementation that increases citric acid cycle intermediates via anaplerosis may have therapeutic advantages over traditional medium-chain triglyceride (MCT) treatment of long-chain fatty acid oxidation disorders (LC-FAODs) but controlled trials have not been reported. The goal of our study was to compare the effects of triheptanoin (C7), an anaplerotic seven-carbon fatty acid triglyceride, to trioctanoin (C8), an eight-carbon fatty acid triglyceride, in patients with LC-FAODs...
November 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28689308/triheptanoin-a-rescue-therapy-for-cardiogenic-shock-in-carnitine-acylcarnitine-translocase-deficiency
#5
Sidharth Mahapatra, Amitha Ananth, Nancy Baugh, Mihaela Damian, Gregory M Enns
Carnitine-acylcarnitine translocase (CACT) deficiency is a rare long-chain fatty acid oxidation disorder (LC-FAOD) with high mortality due to cardiomyopathy or lethal arrhythmia. Triheptanoin (UX007), an investigational drug composed of synthetic medium odd-chain triglycerides, is a novel therapy in development for LC-FAOD patients. However, cases of its safe and efficacious use to reverse severe heart failure in CACT deficiency are limited. Here, we present a detailed report of an infant with CACT deficiency admitted in metabolic crisis that progressed into severe cardiogenic shock who was successfully treated by triheptanoin...
July 9, 2017: JIMD Reports
https://www.readbyqxmd.com/read/28688166/triheptanoin-for-the-treatment-of-brain-energy-deficit-a-14-year-experience
#6
REVIEW
Fanny Mochel
Triheptanoin is an odd-chain triglyceride with anaplerotic properties-that is, replenishing the pool of metabolic intermediates in the Krebs cycle. Unlike even-chain fatty acids metabolized to acetyl-CoA only, triheptanoin can indeed provide both acetyl-CoA and propionyl-CoA, two key carbon sources for the Krebs cycle. Triheptanoin was initially used in patients with long-chain fatty acid oxidation disorders. The first demonstration of the possible benefit of triheptanoin for brain energy deficit came from a patient with pyruvate carboxylase deficiency, a severe metabolic disease that affects anaplerosis in the brain...
July 8, 2017: Journal of Neuroscience Research
https://www.readbyqxmd.com/read/28630259/triacylglycerol-mimetics-regulate-membrane-interactions-of-glycogen-branching-enzyme-implications-for-therapy
#7
Rafael Alvarez, Jesús Casas, David J López, Maitane Ibarguren, Ariadna Suari-Rivera, Silvia Terés, Francisca Guardiola-Serrano, Alexander Lossos, Xavier Busquets, Or Kakhlon, Pablo V Escribá
Adult polyglucosan body disease (APBD) is a neurological disorder characterized by adult-onset neurogenic bladder, spasticity, weakness, and sensory loss. The disease is caused by aberrant glycogen branching enzyme (GBE) (GBE1Y329S) yielding less branched, globular, and soluble glycogen, which tends to aggregate. We explore here whether, despite being a soluble enzyme, GBE1 activity is regulated by protein-membrane interactions. Because soluble proteins can contact a wide variety of cell membranes, we investigated the interactions of purified WT and GBE1Y329S proteins with different types of model membranes (liposomes)...
August 2017: Journal of Lipid Research
https://www.readbyqxmd.com/read/28457799/dietary-treatment-of-fatty-liver-high-dietary-protein-content-has-an-antisteatotic-and-antiobesogenic-effect-in-mice
#8
Sonia C Garcia Caraballo, Tine M Comhair, Cornelis H C Dejong, Wouter H Lamers, S Eleonore Koehler
Few studies have assessed the effect of changing ratios of dietary macronutrients on fat accumulation in adipose tissue and organs such as the liver in a 3×n(n≥3) factorial design. We investigated the effects of 7 diets from a single manufacturer containing 11-58en% protein (casein), 0-81en% carbohydrates (CHO; sucrose, maltrodextrin-10 and corn starch), and 8-42en% fat (triheptanoin, olive oil or cocoa butter) in C57BL/6J mice, a good model for diet-induced obesity and fatty liver. The diets were fed for 3weeks to wild-type and hyperlipidemic male and female mice...
April 28, 2017: Biochimica et Biophysica Acta
https://www.readbyqxmd.com/read/28381467/hepatic-ketogenesis-induced-by-middle-cerebral-artery-occlusion-in-mice
#9
Konrad Koch, Dirk Berressem, Jan Konietzka, Anna Thinnes, Gunter P Eckert, Jochen Klein
BACKGROUND: Ketone bodies are known to substitute for glucose as brain fuel when glucose availability is low. Ketogenic diets have been described as neuroprotective. Similar data have been reported for triheptanoin, a fatty oil and anaplerotic compound. In this study, we monitored the changes of energy metabolites in liver, blood, and brain after transient brain ischemia to test for ketone body formation induced by experimental stroke. METHODS AND RESULTS: Mice were fed a standard carbohydrate-rich diet or 2 fat-rich diets, 1 enriched in triheptanoin and 1 in soybean oil...
April 5, 2017: Journal of the American Heart Association
https://www.readbyqxmd.com/read/27884962/triheptanoin-long-term-effects-in-the-very-long-chain-acyl-coa-dehydrogenase-deficient-mouse
#10
Sara Tucci, Ulrich Floegel, Frauke Beermann, Sidney Behringer, Ute Spiekerkoetter
A rather new approach in the treatment of long-chain fatty acid oxidation disorders is represented by triheptanoin, a triglyceride with three medium-odd-chain heptanoic acids (C7), due to its anaplerotic potential. We here investigate the effects of a 1-year triheptanoin-based diet on the clinical phenotype of very long-chain-acyl-CoA-dehydrogenase-deficient (VLCAD(-/-)) mice. The cardiac function was assessed in VLCAD(-/-) mice by in vivo MRI. Metabolic adaptations were identified by the expression of genes regulating energy metabolism and anaplerotic processes using real-time PCR, and the results were correlated with the measurement of the glycolytic enzymes pyruvate dehydrogenase and pyruvate kinase...
January 2017: Journal of Lipid Research
https://www.readbyqxmd.com/read/27868154/alternative-fuels-in-epilepsy-and-amyotrophic-lateral-sclerosis
#11
Tesfaye W Tefera, Kah Ni Tan, Tanya S McDonald, Karin Borges
This review summarises the recent findings on metabolic treatments for epilepsy and Amyotrophic Lateral Sclerosis (ALS) in honour of Professor Ursula Sonnewald. The metabolic impairments in rodent models of these disorders as well as affected patients are being discussed. In both epilepsy and ALS, there are defects in glucose uptake and reduced tricarboxylic acid (TCA) cycling, at least in part due to reduced amounts of C4 TCA cycle intermediates. In addition there are impairments in glycolysis in ALS. A reduction in glucose uptake can be addressed by providing the brain with alternative fuels, such as ketones or medium-chain triglycerides...
November 21, 2016: Neurochemical Research
https://www.readbyqxmd.com/read/27590926/triheptanoin-treatment-in-patients-with-pediatric-cardiomyopathy-associated-with-long-chain-fatty-acid-oxidation-disorders
#12
J Vockley, J Charrow, J Ganesh, M Eswara, G A Diaz, E McCracken, R Conway, G M Enns, J Starr, R Wang, J E Abdenur, J Sanchez-de-Toledo, D L Marsden
Long-chain fatty acid oxidation disorders (LC-FAOD) can cause cardiac hypertrophy and cardiomyopathy, often presenting in infancy, typically leading to death or heart transplant despite ongoing treatment. Previous data on triheptanoin treatment of cardiomyopathy in LC-FAOD suggested a clinical benefit on heart function during acute failure. An additional series of LC-FAOD patients with critical emergencies associated with cardiomyopathy was treated with triheptanoin under emergency treatment or compassionate use protocols...
November 2016: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/27564703/triheptanoin-protects-motor-neurons-and-delays-the-onset-of-motor-symptoms-in-a-mouse-model-of-amyotrophic-lateral-sclerosis
#13
Tesfaye W Tefera, Yide Wong, Mallory E Barkl-Luke, Shyuan T Ngo, Nicola K Thomas, Tanya S McDonald, Karin Borges
There is increasing evidence that energy metabolism is disturbed in Amyotrophic Lateral Sclerosis (ALS) patients and animal models. Treatment with triheptanoin, the triglyceride of heptanoate, is a promising approach to provide alternative fuel to improve oxidative phosphorylation and aid ATP generation. Heptanoate can be metabolized to propionyl-CoA, which after carboxylation can produce succinyl-CoA and thereby re-fill the tricarboxylic acid (TCA) cycle (anaplerosis). Here we tested the hypothesis that treatment with triheptanoin prevents motor neuron loss and delays the onset of disease symptoms in female mice overexpressing the mutant human SOD1G93A (hSOD1G93A) gene...
2016: PloS One
https://www.readbyqxmd.com/read/26778339/the-odd-carbon-medium-chain-fatty-triglyceride-triheptanoin-does-not-reduce-hepatic-steatosis
#14
Tine M Comhair, Sonia C Garcia Caraballo, Cornelis H C Dejong, Wouter H Lamers, S Eleonore Koehler
BACKGROUND & AIMS: Non-alcoholic fatty-liver disease (NAFLD) is the hepatic manifestation of the metabolic syndrome. Previously, we showed that a high-protein diet minimized diet-induced development of fatty liver and even reversed pre-existing steatosis. A high-protein diet leads to amino-acid catabolism, which in turn causes anaplerosis of the tricarboxylic-acid (TCA) cycle. Therefore, we hypothesized that anaplerosis of the TCA cycle could be responsible for the high-protein diet-induced improvement of NAFLD by channeling amino acids into the TCA cycle...
December 8, 2015: Clinical Nutrition: Official Journal of the European Society of Parenteral and Enteral Nutrition
https://www.readbyqxmd.com/read/26547562/anaplerotic-treatment-of-long-chain-fat-oxidation-disorders-with-triheptanoin-review-of-15-years-experience
#15
Charles R Roe, Henri Brunengraber
BACKGROUND: The treatment of long-chain mitochondrial β-oxidation disorders (LC-FOD) with a low fat-high carbohydrate diet, a diet rich in medium-even-chain triglycerides (MCT), or a combination of both has been associated with high morbidity and mortality for decades. The pathological tableau appears to be caused by energy deficiency resulting from reduced availability of citric acid cycle (CAC) intermediates required for optimal oxidation of acetyl-CoA. This hypothesis was investigated by diet therapy with carnitine and anaplerotic triheptanoin (TH)...
December 2015: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/26536893/triheptanoin-dramatically-reduces-paroxysmal-motor-disorder-in-patients-with-glut1-deficiency
#16
Fanny Mochel, Elodie Hainque, Domitille Gras, Isaac M Adanyeguh, Samantha Caillet, Bénédicte Héron, Agathe Roubertie, Elsa Kaphan, Romain Valabregue, Daisy Rinaldi, Sandrine Vuillaumier, Raphael Schiffmann, Chris Ottolenghi, Jean-Yves Hogrel, Laurent Servais, Emmanuel Roze
OBJECTIVE: On the basis of our previous work with triheptanoin, which provides key substrates to the Krebs cycle in the brain, we wished to assess its therapeutic effect in patients with glucose transporter type 1 deficiency syndrome (GLUT1-DS) who objected to or did not tolerate ketogenic diets. METHODS: We performed an open-label pilot study with three phases of 2 months each (baseline, treatment and withdrawal) in eight patients with GLUT1-DS (7-47 years old) with non-epileptic paroxysmal manifestations...
May 2016: Journal of Neurology, Neurosurgery, and Psychiatry
https://www.readbyqxmd.com/read/26433381/modification-of-astrocyte-metabolism-as-an-approach-to-the-treatment-of-epilepsy-triheptanoin-and-acetyl-l-carnitine
#17
REVIEW
Mussie Ghezu Hadera, Tanya McDonald, Olav B Smeland, Tore W Meisingset, Haytham Eloqayli, Saied Jaradat, Karin Borges, Ursula Sonnewald
Epilepsy is a severe neurological disorder characterized by altered electrical activity in the brain. Important pathophysiological mechanisms include disturbed metabolism and homeostasis of major excitatory and inhibitory neurotransmitters, glutamate and GABA. Current drug treatments are largely aimed at decreasing neuronal excitability and thereby preventing the occurrence of seizures. However, many patients are refractory to treatment and side effects are frequent. Temporal lobe epilepsy (TLE) is the most common type of drug-resistant epilepsy in adults...
February 2016: Neurochemical Research
https://www.readbyqxmd.com/read/26284828/de%C3%A2-novo-fatty-acid-biosynthesis-and-elongation-in-very-long-chain-acyl-coa-dehydrogenase-deficient-mice-supplemented-with-odd-or-even-medium-chain-fatty-acids
#18
Sara Tucci, Sidney Behringer, Ute Spiekerkoetter
An even medium-chain triglyceride (MCT)-based diet is the mainstay of treatment in very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency (VLCADD). Previous studies with magnetic resonance spectroscopy have shown an impact of MCT on the average fatty acid chain length in abdominal fat. We therefore assume that medium-chain fatty acids (MCFAs) are elongated and accumulate in tissue as long-chain fatty acids. In this study, we explored the hepatic effects of long-term supplementation with MCT or triheptanoin, an odd-chain C7-based triglyceride, in wild-type and VLCAD-deficient (VLCAD(-/-) ) mice after 1 year of supplementation as compared with a control diet...
November 2015: FEBS Journal
https://www.readbyqxmd.com/read/26262726/hepatic-fatty-acid-profile-of-rats-fed-a-triheptanoin-based-ketogenic-diet
#19
Ingrid Sofia Vieira de Melo, Terezinha Da Rocha Ataide, Suzana Lima de Oliveira, Nassib Bezerra Bueno, Johnnatan Duarte de Freitas, Antônio Euzébio Goulart Sant'Ana
OBJECTIVE: the aim of this study was to evaluate the influence of consumption of a ketogenic diet supplemented with triheptanoin, a medium-chain anaplerotic triacylglycerol, on the liver fatty acid profile of Wistar rats. METHODS: three groups of male Wistar rats (n = 10) were submitted to an AIN-93 control diet, a triheptanoin- based ketogenic diet, or a soybean oil-based ketogenic diet for 60 days. Excised livers were subjected to lipid extraction and methylation to obtain fatty acids methyl esters, which were subjected to gas chromatography- mass spectrometry...
July 1, 2015: Nutrición Hospitalaria: Organo Oficial de la Sociedad Española de Nutrición Parenteral y Enteral
https://www.readbyqxmd.com/read/26209001/triheptanoin-alleviates-ventricular-hypertrophy-and-improves-myocardial-glucose-oxidation-in-rats-with-pressure-overload
#20
T Dung Nguyen, Yasushige Shingu, Paulo A Amorim, Michael Schwarzer, Torsten Doenst
OBJECTIVE: Cardiac hypertrophy is characterized by changes in substrate utilization and activity of the Krebs cycle. We assessed the effects of triheptanoin, an odd-chain fat that might support the Krebs cycle, on cardiac metabolism and function in a model of cardiac hypertrophy. METHODS AND RESULTS: Rats were subjected to aortic banding (AoB) to induce pressure overload (PO). Starting at 1 week after AoB, rats were blindly fed a control diet or a special diet containing triheptanoin at 7% (T7 group) or 30% (T30 group) of total energy value...
November 2015: Journal of Cardiac Failure
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