立场声明:罕见疾病的新兴基因疗法

Journal of the International Child Neurology Association Pub Date : 2019-07-25 DOI:10.17724/JICNA.2019.172

P. Samia, A. Kirton, R. Dale, S. Tenembaum, C. Triki, A. Hegde, R. Idro, E. Kija, J. Wilmshurst, I. Tein, H. Topaloğlu

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引用次数: 1

摘要

新兴的昂贵的罕见疾病基因疗法不能切实解决全球疾病负担。利益攸关方必须开发新的途径，确保安全、公平和可持续地提供此类疗法。

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Position Statement:Emerging genetic therapies for rare disorders

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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来源期刊

Journal of the International Child Neurology Association

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