Lessons learnt from existing gene therapy trials

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As Gene Therapy is relatively new to the Duchenne community we are keen to learn from experiences in other diseases. This downloadable PDF covers some lessons we have learnt from existing Gene Therapy trials regarding pharmacy, risk assessment, R&D and costing. The document has been drafted by Newcastle, reviewed by GOSH and approved by the chair of the Trust Genetic Modification Safety Committee.  

Link to PDF here.