Deleted Tweet from Scott Gottlieb, R-D.C.
Deleted Tweet from Scott Gottlieb, M.D.
SteveFDA (R-D.C.)
@SGottliebFDA
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FDA’s new draft guidance clarifies evidence necessary to demonstrate effectiveness of drugs for slowly progressive, low-prevalence rare diseases associated with substrate deposition and are caused by single enzyme defects (e.g. Gaucher, Pompe, Tay Sachs): https://t.co/yLdDPgAGY3
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