Position Statement:Emerging genetic therapies for rare disorders

Authors :: Silvia Tenembaum
Russell C. Dale
Richard Idro
Pauline Samia
Ingrid Tein
Adam Kirton
Chahnez Triki
Jo M. Wilmshurst
Edward Kija
Haluk Topaloglu
Anaita Udwadia Hegde
Source :: Journal of the International Child Neurology Association.
Publication Year :: 2019
Publisher :: The International Child Neurology Association (ICNA), 2019.
Abstract: Emerging genetic therapies for rare disorders at high cost, cannot realistically address the global burden of disease. Stakeholders must develop new pathways to ensure safe, fair and sustainable provision of such therapies.