Practical and ethical challenges in treatment of paediatric neurogenetic disorders
There has in recent years been a rapid increase in the number and complexity of clinical trials and novel therapies for neurogenetic conditions. Many of these conditions are individually rare, but their impact upon affected children and their families may be very severe. While the increasing awareness and availability of new treatments brings great hope and excitement for all involved in care of these children, it also presents significant challenges for clinicians and for patients and their families.