Dmd – therapy

Duchenne muscular dystrophy (DMD) is a fatal, rare, X-linked disease characterized by progressive muscle weakness. Approximately 10–15% of DMD cases are caused by a nonsense mutation (nmDMD) in the dystrophin gene, resulting in the absence of functional dystrophin. Ataluren (10, 10, 20 mg/kg [morning, midday, and evening]) targets the underlying cause of nmDMD, enabling the formation of full-length, dystrophin. Study 019 was a phase 3, long-term safety study that enrolled nmDMD patients from prior ataluren clinical trials (NCT01557400) (N=94).