Ataluren is indicated for the treatment of Duchenne muscular dystrophy that results from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older in the European Member States and Iceland, Liechtenstein, Norway, Great Britain, Northern Ireland, Kazakhstan, Israel, Republic of Korea, Belarus, Russia, and Brazil, and aged 5 years and older in Chile, the Kingdom of Saudi Arabia, and Ukraine (under special state registration). The gene discussed is DMD; the disease is Duchenne muscular dystrophy.