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Draft Guide on Rare Disease Gene Therapies
07/12/2018
 

Federal Register notice: FDA has made available a draft document entitled “Human Gene Therapy for Rare Diseases; Draft Guidance for Industry.” The document provides recommendations on designing clinical development programs for such products where there may be limited study population size and potential feasibility and safety issues, as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the gene therapy itself. To view the notice, click here.


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