Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy

Recently, the Food and Drug Administration granted accelerated approvals for four exon skipping therapies –Eteplirsen, Golodirsen, Viltolarsen, and Casimersen –for Duchenne Muscular Dystrophy (DMD). However, these treatments have only demonstrated variable and largely sub-therapeutic levels of resto...

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Detalles Bibliográficos
Autores principales: Novak, James S., Spathis, Rita, Dang, Utkarsh J., Fiorillo, Alyson A., Hindupur, Ravi, Tully, Christopher B., Mázala, Davi A.G., Canessa, Emily, Brown, Kristy J., Partridge, Terence A., Hathout, Yetrib, Nagaraju, Kanneboyina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8673539/
https://www.ncbi.nlm.nih.gov/pubmed/34569969
http://dx.doi.org/10.3233/JND-210696

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