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Ataluren for drug‐resistant epilepsy in nonsense variant‐mediated Dravet syndrome and CDKL5 deficiency disorder

OBJECTIVE: Ataluren is a compound that reads through premature stop codons and increases protein expression by increasing translation without modifying transcription or mRNA stability. We investigated the safety and efficacy of ataluren in children with nonsense variants causing Dravet Syndrome (DS)...

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Detalles Bibliográficos
Autores principales:	Devinsky, Orrin, King, LaToya, Bluvstein, Judith, Friedman, Daniel
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7951093/ https://www.ncbi.nlm.nih.gov/pubmed/33538404 http://dx.doi.org/10.1002/acn3.51306

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