The 2024 FA Symposium gave me hope for the future of treatments

As I headed west down the familiar highway from Pennsylvania to Ohio, my mind spun as my eyes took in the autumn hues around me. After two amazing days at the 2024 Friedreich’s Ataxia Symposium, I welcomed the long ride home in quiet.

Usually, my drives to Philadelphia involve my 12-year-old daughter, Amelia, sitting in the back seat. She has Friedreich’s ataxia (FA), and we must cross state lines to see her specialists. Today, all I saw was my luggage taking up that space. Though my mind needed the quiet, my heart missed both of my girls.

I own my struggle with understanding the science behind FA research and the treatment pipeline. I joke that I attend these meetings with Google handy because I need to look up so many things. But I didn’t need outside assistance to impress upon me how much promise we’re seeing in new and developing treatments for FA.

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Two sections of the symposium’s agenda on Monday, Oct. 7, were devoted to panel discussions with pharmaceutical companies committed to helping slow, stop, and reverse the symptoms of FA. If you missed attending the symposium in person or on Zoom, I’d encourage you to take the time to watch it on the Friedreich’s Ataxia Research Alliance’s YouTube page when it’s released. The information was impressive.

Pharmaceutical company Biogen discussed the second enrollment in its pediatric Phase 1 trial for Skyclarys (omaveloxolone), a critical step in ensuring pediatric access to the FA community’s one approved drug. Larimar Therapeutics announced that it’ll begin enrolling pediatric cohorts in a study on the investigational drug nomlabofusp in four to six weeks.

Despite all the positives, I still find myself struggling with the pace of research. I’m not a patient person; I want access to treatment for my child now. It’s so hard being so close to possible treatments and watching Amelia’s mobility and health continue to fade. I know this ask isn’t reasonable, but I cannot help how I feel.

Thinking about that, I tapped the brakes of my car as traffic slowed on roads lined with orange barrels. Highway lanes merged to support road construction, ruining my chances of getting home before school let out for both of my girls. I chuckled with the realization that I’ve driven this highway almost a dozen times over the past three and a half years, and there always seems to be construction.

As I inched past workers controlling heavy machinery, my mind skipped from the presentations at the symposium to a notification I’d received first thing in the morning. PTC Therapeutics announced “statistically significant results on primary endpoint of long-term study analyses” for its investigational drug vatiquinone. The company further reported that it would move forward with submitting a New Drug Application to the United States Food and Drug Administration by the end of the year. This application would help address “the high unmet need, especially in the pediatric population.”

I felt the smile grow across my face as I dreamed about the possibility of having two approved medications for what used to be an untreatable disease. Tears welled in my eyes as I thought about all the individuals who worked so hard paving this path over so many years and made so many sacrifices.

After several miles of staring at red brake lights, traffic began to clear. The orange cones gave way to a smoothly surfaced open highway. Beaming at the road ahead, I hit the gas.

Note: 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. The opinions expressed in this column are not those of Friedreich’s Ataxia News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Friedreich’s ataxia.