A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood

A randomized, controlled, double-blind, crossover trial of triheptanoin in alternating hemiplegia of childhood

Abstract 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 cros...

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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: BMC 2017-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Alternating hemiplegia of childhood
Triheptanoin
Crossover trial
Online Access:http://link.springer.com/article/10.1186/s13023-017-0713-2

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