Name: Bernard Ravina
Where do you work? Praxis Precision Medicines, Cambridge, MA
How long have you been working on FA and who was the first fellow FA researcher you met? I have been working on FA for about 20 years. I started working on clinical scale validation studies with Kurt Fischbeck and team at NINDS/NIH (Neurogenetics branch).
What got you interested in FA research? I was generally interested in mitochondrial disorders and therapeutics for mitochondrial diseases. I thought FA was a great example and one where we were more likely to find treatments. Then I met the team of researchers and the family community, and I was hooked.
What question or challenge were you setting out to address when you started this work? My initial involvement was in preparation for trials of Idebenone and other antioxidants.
What research topics or questions are you currently focused on? My work in FA has always had a focus on therapeutics and enabling clinical trials. After I moved to the University of Rochester, we became the coordinating center for the natural history study (FACOMS). When I moved to the biotechnology field, I worked on AAV gene therapy at Voyager Therapeutics. My current company does not focus on FA, but I remain active with FARA.
What do you hope to achieve or what excites you in FA research? The idea of finding a meaningful treatment that slows the overall course or an important aspect like cardiomyopathy continues to drive my interest. I believe this can be done although the pace of research is never predictable.
If you have met someone living with FA, please tell us about that interaction. Did it have an impact on your work? I have met so many people in the community, people with FA, their families, and their friends. There is something uniquely warm and genuine about the way the FA community works together to make progress. It keeps me coming back.
You serve voluntarily on FARA's Scientific Advisory Board. Please tell us what you see as FARA's key role in the research process. FARA does a great job engaging the overall FA research community, raising interest, supporting research tools from preclinical to clinical, and bringing new people to the field. FARA's work has led to a remarkable interest in FA and many promising preclinical and clinical programs.