DUCHENNE DISEASE: COMPLEXITIES IN THE USE OF ATALUREN IN SPAIN.

Duchenne muscular dystrophy is a rare genetic disease which affects a small proportion of the population and for which there is currently only one authorized treatment; ataluren. Since this drug was approved, aspects such as its real efficacy, its financing and its use in the different countries of Europe and also in the different regions of Spain have been questioned. In our country, ataluren is not a financed drug, which prevents the public health system from treating affected children. This situation has generated a profound debate, in which ethical aspects such as equity and justice are involved, as well as some legal principles such as the protection of minors.