Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength

Ebrahimi-Fakhari D, Dillmann U, Flotats-Bastardas M, et al. Off-Label Use of Ataluren in Four Non-ambulatory Patients With Nonsense Mutation Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength. Frontiers in pediatrics 2018;6:316   Abstract About 15% of Duchenne muscular dystrophy (DMD) cases are caused by point mutations leading to premature stop codons […]