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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.

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How to Cite
Samia, P., Kirton, A., Dale, R., Tenembaum, S., Triki, C. C., Hegde, A., Idro, R., Kija, E., Wilmshurst, J. M., Tein, I., & Topaloğlu, H. (2019). Position Statement:Emerging genetic therapies for rare disorders: Genetic therapies. Journal of the International Child Neurology Association. Retrieved from http://jicna.org/index.php/journal/article/view/172