Personal take on this article:   The study investigates the role of high mobility group box protein 1 (HMGB1) as a potential biomarker for Duchenne muscular dystrophy (DMD) by utilizing RNA sequencing in mouse models and human induced pluripotent stem cell (iPSC)-derived skeletal muscle cells. The findings suggest that HMGB1 is elevated in DMD conditions and can be reduced with gene therapy using microdystrophin. This indicates that HMGB1 …

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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 …

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Personal take on this article: A potential therapy for a specific type of muscular dystrophy (LGMD2B), caused by mutations in the DYSF gene leading to reduced dysferlin protein levels, involves membrane stabilization. Dysferlin is crucial for repairing the membrane of healthy muscle fibers, and its deficit results in chronic muscle inflammation. In this study, the researchers compared the effects of two steroids, prednisolone and vamorolone (VBP15), on repairing dysferlin-deficient muscle …

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