The drug is currently the only treatment available for patients with Duchenne muscular dystrophy with a nonsense mutation. In Italy, Translarna™️ (ataluren) is available since 2014 for patients 5 years and older, and since 2021 for children 2 years and older.
This decision, if confirmed, threatens to diminish the therapeutic options that we have achieved over the past 25 years with our international Duchenne community.
To make our voice heard at the EMA, we have created this website and the video below.