Duchenne muscular dystrophy is a rare genetic disease with only ataluren like pharmaceutical treatment available. This drug received a conditional authorization by the European Medicines Agency (EMA) in 2014, meaning that it was commercially available while waiting for more solid results that demonstrate the efficacy and safety. Currently, the authorization still maintains the ″conditional″ modality, and the actual health benefits of the drug still remain unclear. In Spain, ataluren is not financed by the National Health System. The decision of non-financing has generated a heated debate, especially because in those patients who se treatment were started prior to the non-financing decision the drug has continued being funded. This controversial situation encompasses complex aspects of pharmaceutical and health management related to scientific evidence, the motivations of regulatory agencies and the pharmaceutical industry in the processes of drug investigation and authorization. Furthermore, as the center of the debate, some pillars of bioethics such as justice and equity, as well as certain legal principles, such as the protection of minors, are involved.