Friedreich’s ataxia (FA) is a rare, hereditary disease that affects the nervous system and can lead to severe disability. For years, there has been no cure or treatment available for this condition, leaving patients and their families in constant despair. However, with the recent development of a new drug that shows remarkable promise in treating FA, the future seems bright. So why do patients like me feel afraid? The answer is a complex mix of hope, uncertainty, and fear of the unknown. This article delves deeper into the world of FA, explores the excitement surrounding the new drug, and unpacks the emotions behind the hesitation.
On Rare Disease Day, just before midnight on February 28th, I received the exciting news that the Federal Drug Administration (FDA) had approved the first ever treatment for Friedreich’s ataxia (FA), the rare neuromuscular disorder I have. However, my emotions quickly turned to anxiety and fear about whether the treatment would be accessible in Ireland due to the country’s track record of funding rare disease treatments. I had previously called on the government to establish a funding structure for expensive treatments, as many rare disease patients have to campaign for approval, like cystic fibrosis patients did to gain access to Orkambi. Although the approval of the drug, Omaveloxolone, is a monumental step in the right direction, there is still uncertainty around approval and funding. My hope is that the approval of this drug clears the path for all future treatments to be approved more easily. Time is of the essence for FA patients, and we need the government to implement a better and faster rare disease treatment funding structure.
In conclusion, the prospect of a potential game-changing drug for Friedreich’s ataxia is undoubtedly exciting news for those living with the condition. However, it’s understandable that there may still be some fear and apprehension surrounding the drug’s development and potential long-term effects. It’s crucial that healthcare professionals and researchers continue to prioritize safety and transparency throughout the drug’s testing and approval process. While there may still be some uncertainty about what the future holds, it’s important to remain hopeful and continue to advocate for advancements in the treatment and management of Friedreich’s ataxia.
