Advocating for the Friedreichs Ataxia community

February 26th, 2025

Living with Friedreich's ataxia can present unique challenges, both physically and emotionally. It's crucial for individuals with this rare neuromuscular disease to have access to support and resources to help navigate their journey. Recently our grants and funding have been put at risk. I had the opportunity to travel to Washington D.C. during rare disease week February 24th-28th with my fiance. It was an opportunity to speak to our state representatives about the asks we had as a community for Congress to hear. My ask was, "ensuring steady and robust federal biomedical research funding and public health agency support." Living in East TN my representatives are, Senator Marsha Blackburn, Senator Bill Hagerty, and Rep. Diana Harshbarger. I had never traveled to D.C. before so on top of my first advocating experience, was my first time at the Capitol. I thought D.C. was such a beautiful city. We saw the Lincoln Memorial, The White House, The Capitol, visited the Smithsonian Museum and ate at so many great places. We arrived on the 24th, checked into our hotel and got dinner, the next day on the 25th was the beginning of my advocacy. We attended the Legislative Conference. This event was from 8 am- 5 pm, starting with check-in/group breakfast. Then we got to begin listening to speakers including, Duane Clark, Annie Kennedy (EveryLife Foundation for Rare Diseases), Amy Browser Ph.D. (Creighton University School of Medicine), Jennifer Bernstein (Horizon Government Affairs, EveryLife Foundation Board Member), Kevin Brennan (NCATS Alliance), E. Cartier Esham Ph.D. (Alliance for a Stronger FDA). Switching gear to Neena Nizar, Ph.D. (The Jansen's Foundation), Deanna Portero (Orphan Therapeutics Accelerator), and Jill Wood (Phoenix Nest Inc). We then got a group photo at the atrium stairs, following with a networking lunch, after lunch we split into our states, mine being TN, to prepare for successful meetings. During this time I got to meet 6 amazing individuals, 2 lived with rare disease themselves, and 4 were there advocating for their children. We had to discuss how and what we wanted to address to our representatives. After an hour of getting to know each other we decided who would speak, when, and about what. It was decided that I would do the speaking to Diana Harshbarger since she's my area representative. Later that night we got to go eat with individuals from the FA community. It was so exciting to meet others who live with FA like myself. The next day we had meetings from 10 am- 4 pm to start advocating. I want to share what I said to my representative. Keeping in mind this was my first experience and I had to write this the night before.. "Hi my name is Alexis Baker, I am from Rogersville, TN. I was diagnosed in 2019 with Friedreichs Ataxia, a neurological disease that affects balance and coordination. In 2023 the first FDA treatment got approved, Skyclary's. Without it my progression would be worse then what it is. Skyclary's has given me mobility and the confidence I need to succeed in living the most normal life I can. In 2024 I got engaged, graduated college, and my 1st nephew turned one. I dream of starting my career, getting to walk down the aisle and help raise my nephew. The grants and funding currently in place are crucial for our survival and well being. They support vital research that brings us closer to affective treatments and hopefully a cure. I urge you to continue supporting these grants and funding initiatives because without them could be a death sentence to many of us with a rare disease. Your support can make a difference in the lives of many who struggle with a rare disease. Thank you for your support and consideration." This was my first advocating experience and certainly not my last. If you have the chance yourself, DO IT! Such a wonderful feeling and has such a great impact for everyone with a rare disease.