CSPAN2 Key Capitol Hill Hearings July 11, 2014
Hope. I could tell you stories about looking in china to see theres some tea you might be up uphold about, but that was long ago. I think theres also it really is up to the companies. Fda has always to my knowledge at least in duchenne and other fields been willing to entertain and talk about compassionate use . I think for the Rare Disease Community is talks about and get us back to trial design but in general, trials are designed to test a sub set of patients. In the duchenne committee, the six minute walk test is standard outcome, primary outcome measure. That means a child with duchenne wo...
Source: archive.org
