This makes the potential DMD announcement, if it is that, more interesting"
"Orphan drug trials are generally single arm (no placebo arm), nonrandomized, and open label. Safety Phase 1 trials are not usually required, and Phases 2 and 3 can be combined when the patient population is very low."
https://repository.upenn.edu/cgi/viewcontent.cgi?article=1037&context=ace
Ann: Trading Halt, page-34
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