Duchenne muscular dystrophy, or DMD in short, is a rare and progressive genetic muscle disease caused by mutations in the dystrophin-encoding DMD gene and it occurs primarily in boys. DMD patients lack dystrophin, a critical membrane-stabilizing protein, which leads to muscle degeneration and weakness. The DMD gene contains 79 exons which are pieces of coding DNA that encode the protein dystrophin. Exon-skipping is one of the therapies that can restore the expression of partially functional dystrophin protein. Approximately 8%-10% of DMD patients have mutations amenable to treatment with an exon 53 skipping therapy.
The stock we are bringing to your attention today is that of Wave Life Sciences Ltd. (WVE), which is developing a treatment for boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.
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