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During the course of the webinar, Keefer explained how specialty sites, with deep knowledge on one or certain groups of Rare Diseases, are crucial for further progress in drugdevelopment to progress the number of treatments available to Rare Disease patients and to provide support to those individuals. Headway being made.
In this Q&A, Karen Pinachyan, Head of Medical Affairs Europe at CSL Behring summarises key considerations for gene therapy drugdevelopment and the ideal approach for alleviating economic strain when advancing these modern treatments. 4,5 What is the key consideration drugdevelopers should apply to gene therapies?
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