This year, Acceleron’s ACE-083 drug is expected to complete its phase 2b trial, and Fulcrum is conducting its trial of
the first drug intended to repress DUX4, the gene implicated in causing FSHD. This is a good time to brush up on the vocabulary of clinical trials and the FDA approval process, and learn how to navigate the path to volunteering for trials. Your guide is Rabi Tawil, MD, who is principal investigator for several clinical trials going on at the University of Rochester’s Fields Center for FSHD Research.
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