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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.


rare disorders position statement gene therapy

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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, 1(1).

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