A randomised placebo-controlled, double-blind phase II study to explore the safety, efficacy, and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease and motor symptoms (“KHENERGYC”)

BACKGROUND: METHODS: The KHENERGYC trial will be a phase II, randomised, double-blinded, placebo-controlled (DBPC), parallel-group study in the paediatric population (birth up to and including 17 years). The study will be recruiting 24 patients suffering from motor symptoms due to genetically confir...

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Detalles Bibliográficos
Autores principales: Smeitink, Jan, van Maanen, Rob, de Boer, Lonneke, Ruiterkamp, Gerrit, Renkema, Herma
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9044835/
https://www.ncbi.nlm.nih.gov/pubmed/35477351
http://dx.doi.org/10.1186/s12883-022-02685-3

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