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Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy
Personal take on this article: Researchers studied the turnover of dystrophin and dystroglycan complex (DGC) proteins in mice with Duchenne Muscular Dystrophy (DMD) after receiving exon skipping therapy. Exon skipping therapies, such as Eteplirsen, Golodirsen, Viltolarsen, and Casimersen, have received accelerated approvals from the FDA for DMD. However, these therapies have shown variable and often insufficient levels of restored dystrophin protein in patients, limiting their effectiveness. To understand the …
