Rare paediatric neurological conditions are debilitating diseases with significant impact on the daily lives of children and their families. Most rare diseases have neurological symptoms, and about 90% of those affecting children have major neurological effects limiting the ability to achieve key cognitive and physical development milestones.
To highlight policy priorities for improving care, patient outcomes and quality of life, Economist Impact, sponsored by PTC Therapeutics undertook a project to better understand how these disorders affect the lives of patients and their families. The resulting report ‘Rare paediatric neurological diseases: A focus on Europe’ highlights the difficulties in achieving a timely and accurate diagnosis, as well as the numerous challenges to providing adequate care and support. It concludes that policymakers should work, together with other stakeholders, to develop a rare diseases healthcare ecosystem that is sufficiently resourced and organized to enable even the most complex conditions to be assessed and managed expeditiously.