In a previous column, I wrote about the power of patient advocacy. I’m motivated to embrace advocacy in my everyday life to let the world know that disabilities come in all ages, shapes, and sizes, just like Friedreich’s ataxia (FA). This disease is extremely rare, affecting only 15,000 people worldwide — and I am one of them.
That column was perfect timing. Last week, fellow FA patients Connor Sweeny and Kyle Bryant, members of the Friedreich’s Ataxia Research Alliance (FARA), and I had the opportunity to visit one of the organization’s gene therapy partners, PTC Therapeutics. One hundred people from the company attended, and the event was livestreamed to a global audience. FARA screened the documentary “The Ataxian,” and a Q&A discussion followed.
If you have not seen “The Ataxian,” I suggest you do so. The documentary is about Kyle Bryant and his three friends Sean (who also has FA), John, and Mike, who embark on the Race Across America. Dubbed “the world’s toughest bike race,” it took them over nine days to complete. The documentary offers viewers an insight into what each participant felt upon finishing this incredible journey. The film also looks at the origins of rideATAXIA and features stories from families about how FA affects them.
Having FARA members and researchers come together provided a different perspective of Friedreich’s ataxia and how it affects each patient. Connor, Kyle, and I hosted the Q&A panel and answered some excellent questions relating to symptoms, participation in clinical trials and studies, how FA impacts our daily lives, and more.
Following the Q&A discussion, about half of the members and researchers approached us. Each shook our hands, thanked us for sharing our stories, and told us that it truly makes a difference when they see and hear patients in person.
Just two hours long, the meeting was insightful and motivating for everyone in the room, despite its brevity. During lunch afterward, representatives from PTC Therapeutics thanked patients and FARA members for attending. They said the event marks a new beginning and was a powerful introduction as the company tries to make an impact on the FARA research pipeline. They expressed interest in attending FA-related events, including both grassroots and national ones like rideATAXIA, to build trust with FA patients and their families.
I know the opportunity to speak to a pharmaceutical company working on an FA treatment or cure does not come around often. But when it does, take advantage and share your story. We can make a difference and motivate researchers to continue the remarkable work they do for us. It is a pretty amazing feeling to be in a room of 100 people who want a cure just as much as the FA community does.
Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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