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

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 s...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Hainque, Elodie, Caillet, Samantha, Leroy, Sandrine, Flamand-Roze, Constance, Adanyeguh, Isaac, Charbonnier-Beaupel, Fanny, Retail, Maryvonne, Le Toullec, Benjamin, Atencio, Mariana, Rivaud-Péchoux, Sophie, Brochard, Vanessa, Habarou, Florence, Ottolenghi, Chris, Cormier, Florence, Méneret, Aurélie, Ruiz, Marta, Doulazmi, Mohamed, Roubergue, Anne, Corvol, Jean-Christophe, Vidailhet, Marie, Mochel, Fanny, Roze, Emmanuel
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2017
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5625655/
https://ncbi.nlm.nih.gov/pubmed/28969699
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0713-2
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