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Contributors EH, AM, ER and FM drafted the manuscript for content. FM designed the study. EH, DG, M-PL, MA, MB, CO and FM acquired data. EH and FM analysed/interpreted data. EH performed the statistical analysis.
Funding Ultragenyx Pharmaceutical Inc. provided the investigational drug triheptanoin and funded the study. The research leading to these results has received funding from the programme ‘Investissements d’avenir’ ANR-10-IAIHU-06.
Competing interests FM has a patent on the use of triheptanoin in GLUT1-DS (WO2014093901), which has been issued. AM reports non-financial support from ABBVIE, outside the submitted work. ER reports grants, personal fees and non-financial support from Orkyn, grants, personal fees and non-financial support from Aguettant, grants, personal fees and non-financial support from Merz pharma, grants from Ipsen, personal fees from Medday pharma, personal fees from Retrophin, grants and non-financial support from Elivie, grants from Fondation Desmarest, grants from Fonds de dotation Brou de Laurière, grants from Agence Nationale de la Recherche, grants from AMADYS, grants, personal fees and non-financial support from Everpharma, personal fees and non-financial support from Movement Disorders Society, personal fees from European Academy of Neurology, personal fees from International Association of Parkinsonism and related Disorders, non-financial support from Merck, non-financial support from Dystonia Coalition, non-financial support from Dystonia Medical Research Foundation, outside the submitted work.
Patient consent for publication Not required.
Ethics approval Patients and child’s legal guardian signed a written informed consent for participation in this study sponsored by INSERM and approved by the local ethical committee (ID RCB: 2013-A01300-45).
Provenance and peer review Not commissioned; externally peer reviewed.
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