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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)...
Wedi'i Gadw mewn:
| Cyhoeddwyd yn: | Ann Clin Transl Neurol |
|---|---|
| Prif Awduron: | , , , |
| Fformat: | Artigo |
| Iaith: | Inglês |
| Cyhoeddwyd: |
John Wiley and Sons Inc.
2021
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| Pynciau: | |
| Mynediad Ar-lein: | 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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