Triheptanoin dramatically reduces paroxysmal motor disorder in patients with GLUT1 deficiency
Triheptanoin dramatically reduces paroxysmal motor disorder in patients with GLUT1 deficiency
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Mochel, Fanny , Hainque, Elodie , Gras, Domitille , Adanyeguh, Isaac M , Caillet, Samantha , Héron, Bénédicte , Roubertie, Agathe , Kaphan, Elsa , Valabregue, Romain , Rinaldi, Daisy , Vuillaumier, Sandrine , Schiffmann, Raphael , Ottolenghi, Chris , Hogrel, Jean-Yves , Servais, Laurent and Roze, Emmanuel
Publisher
England: BMJ Publishing Group LTD
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English
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England: BMJ Publishing Group LTD
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ObjectiveOn 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.MethodsWe performed an open-label pilot study with thre...
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Triheptanoin dramatically reduces paroxysmal motor disorder in patients with GLUT1 deficiency
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4853553
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4853553
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ISSN
0022-3050
E-ISSN
1468-330X
DOI
10.1136/jnnp-2015-311475