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

Viac o knihe

Duchenne muscular dystrophy, a rare genetic disorder, currently has only one authorized treatment: ataluren. However, its efficacy, financing, and availability vary across Europe and Spain, where it is not covered by public health systems, leaving affected children without access to treatment. This situation has sparked a significant debate encompassing ethical issues of equity and justice, alongside legal principles concerning the protection of minors, highlighting the complexities surrounding healthcare access for rare diseases.