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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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Copyright (c) 2019 Pauline Samia, Haluk Topaloglu, Adam Kirton, Russell Dale, Silvia Tenembaum, Chahnez Charfi Triki, Anaita Hegde, Richard Idro, Edward Kija
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