Advocating for orphan diseases in neurology

Abstract

Patients with rare diseases face several common problems caused by the rarity of their diseases. Among many other issues, these include the often long-delayed diagnosis, the limited knowledge and dissemination of knowledge on the diseases, the lack of specific therapies, and the inadequacies of healthcare systems to deal with rare, complex disorders. In recent years patient advocacy organization have emerged for many different rare diseases as a consequence of these unmet needs. They have started to address these issues by many different advocacy actions such as education, political advocacy, and measures to facilitate research and drug development. Successful examples have shown that such efforts can bring real advances for some rare diseases.