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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