Disease-Modifying Effects of Edasalonexent, an NF- κB Inhibitor, in Young Boys with Duchenne Muscular Dystrophy: Results of the MoveDMD Phase 2 and Open Label Extension Trial

Duchenne muscular dystrophy (DMD) is the most common genetic neuromuscular disease of childhood, affecting 1 in 3,500-6000 male births [1, 2]. DMD manifests clinically as progressive symmetric muscle weakness and degeneration with loss of contractile function and is uniformly fatal with death most often resulting from cardio-pulmonary complications in early- to mid-adulthood [3]. DMD is caused by a variety of mutations in the DMD gene, which encodes dystrophin, a critical protein linker to the sarcolemma membrane complex.

Source: Neuromuscular Disorders - February 3, 2021 Category: Neurology Authors: Richard S. Finkel, Erika Finanger, Krista Vandenborne, H Lee Sweeney, Gihan Tennekoon, Perry B. Shieh, Rebecca Willcocks, Glenn Walter, William D. Rooney, Sean C. Forbes, William T. Triplett, Sabrina W. Yum, Maria Mancini, James MacDougall, Angelika Fretz Source Type: research