La distrofia muscular de Duchenne es una enfermedad genética rara con un único tratamiento autorizado, ataluren. Su eficacia, financiación y uso en Europa y España han sido objeto de debate, especialmente porque en España no está financiado, lo que plantea cuestiones éticas sobre equidad y justicia en el tratamiento de los niños afectados.
Pharmaceutical care and bioethical principles related to the case.
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.