Omigapil Treatment Decreases Fibrosis and Improves Respiratory Rate in dy.sup.2J Mouse Model of Congenital Muscular Dystrophy

Congenital muscular dystrophy is a distinct group of diseases presenting with weakness in infancy or childhood and no current therapy. One form, MDC1A, is the result of laminin alpha-2 deficiency and results in significant weakness, respiratory insufficiency and early death. Modification of apoptosis is one potential pathway for therapy in these patients. dy.sup.2J mice were treated with vehicle, 0.1 mg/kg or 1 mg/kg of omigapil daily via oral gavage over 17.5 weeks. Untreated age matched BL6 mice were used as controls. Functional, behavioral and histological measurements were collected. dy.sup.2J mice treated with omigapil showed improved respiratory rates compared to vehicle treated dy.sup.2J mice (396 to 402 vs. 371 breaths per minute, p