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:   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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