@article{Samia_Kirton_Dale_Tenembaum_Triki_Hegde_Idro_Kija_Wilmshurst_Tein_Topaloğlu_2019, title={Position Statement:Emerging genetic therapies for rare disorders: Genetic therapies}, volume={1}, url={https://jicna.org/index.php/journal/article/view/jicna-2019-172}, DOI={10.17724/jicna.2019.172}, abstractNote={<p>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.</p>}, number={1}, journal={Journal of the International Child Neurology Association}, author={Samia, Pauline and Kirton, Adam and Dale, Russell and Tenembaum, Silvia and Triki, Chahnez Charfi and Hegde, Anaita and Idro, Richard and Kija, Edward and Wilmshurst, Jo M and Tein, Ingrid and Topaloğlu, Haluk}, year={2019}, month={Jul.} }