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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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Dades bibliogràfiques
Publicat a:Ann Clin Transl Neurol
Autors principals: Devinsky, Orrin, King, LaToya, Bluvstein, Judith, Friedman, Daniel
Format: Artigo
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7951093/
https://ncbi.nlm.nih.gov/pubmed/33538404
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51306
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