TRANSLARNA (ataluren), RNA interference agent

Minor improvement in the management of Duchenne muscular dystrophy

• TRANSLARNA has Marketing Authorisation in the treatment of Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.
• Available data suggest a potential benefit, poorly demonstrated and modest, in terms of slowing the loss of ambulation in some children with Duchenne muscular dystrophy, with acceptable safety.
• Efficacy has not been demonstrated in patients who have lost ambulation.