Synopsis

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. Novel neurogenetic therapies are often very expensive, their administration can be complex, and inequities or perceived inequities in access to these treatments can be very challenging for parents. We will present several case studies of children with neurogenetic disorders receiving newer therapies at RCH, and discuss the ethical challenges encountered in their care.

Speakers

Professor Monique Ryan is director of the Neurology Department at RCH and head of the RCH Neuromuscular Research Program

Professor John Massie is the Clinical Director of the Children’s Bioethics Centre at The Royal Children’s Hospital (RCH) Melbourne. John has published numerous papers, letters and book chapters on ethical issues relating to paediatric medicine as well papers on screening for inherited diseases, newborn screening, cystic fibrosis and technology dependent children. John is the producer and host of Essential Ethics, the CBC podcast channel presenting thoughtful discussion on the ethical dilemmas that arise when caring for sick children. John is also a paediatric respiratory physician at RCH, looking after children with cystic fibrosis and those requiring ventilator support.  John is a Clinical Professor at the University of Melbourne and Research Fellow at the Murdoch Children’s Research Institute.

Professor Lynn Gillam is a clinical ethicist at RCH, and Academic Director of the Children’s Bioethics Centre

Dr Heidi Peters is the Clinical Lead, Metabolic Services