We’re pleased to share with you this video recording from the FSHD Society’s webinar, “ABC’s of Clinical Trials,” given on December 7, 2019. Our webinar speaker is Rabi Tawil MD, a foremost authority on FSH muscular dystrophy. Dr. Tawil is director of the Fields Center for FSHD Research at the University of Rochester in Rochester, NY.
In addition to being an outstanding clinician caring for individuals with FSHD, he has led the way in clinical research as a principal investigator for the Clinical Trial Research Network, the U.S. FSHD registry, the ReSolve study, and many clinical trials for FSHD including, most recently, of Acceleron’s ACE-083 and Fulcrum’s ReDUX4 trial of losmapimod.
In this webinar, Dr. Tawil explains the clinical trial process by which potential therapies are validated and approved by the FDA. You’ll learn what Phase 1, Phase 2, and Phase 3 trials are. He lists questions for patients to ask themselves when considering whether to volunteer for a trial, and describes some of the special considerations the FDA gives for orphan indications like FSHD. We thank Dr. Tawil for his time and erudition, and we thank the webinar audience for asking many interesting questions!
I would like to publicly thank the FSH Society and researchers such as Dr. Rabi Tawil for their years of compassionate, tireless work toward finding a cure for FSHD. I have been a member of this community for over 35 years. The dedication of the researchers and all those at the society often fuels my spirit and gives me hope on dark days.
The sincerest of thank you‘s to everyone!
Anne Harland
Canadian FSHD Network
Is it possible for Dr. Tawil to concisely summarize where we are today with respect to FSH research?
Which “avenue“ of controlling DUX 4 appears at this point to hold the greatest promise, be the most viable?
Is it likely that research will result in a treatment but not a cure? i.e. stop further deterioration but not repair “wasted muscle”?
Anne Harland
Canadian FSHD Network