Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene on the X chromosome. These mutations disrupt the production of the dystrophin protein, crucial for muscle stability and function. This review focuses on exon-skipping therapy, which uses genetic techniques to restore partial dystrophin production, converting the severe DMD phenotype to a milder Becker Muscular Dystrophy (BMD)-like condition. Exon-skipping therapy involves antisense oligonucleotides (AONs) that …

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    Personal take on this article:   This document reports on the findings from a Phase 2b clinical trial that evaluated the effects of vamorolone, a novel steroid, and prednisone on adrenal suppression in boys with Duchenne muscular dystrophy (DMD). The study involved 121 steroid-naive boys aged 4 to under 7 years with genetically confirmed DMD, and assessed adrenal function using morning cortisol levels and ACTH-stimulated cortisol levels. Adrenal …

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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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