Washington, D.C. — Friedreich's ataxia patients and families have more reason than ever to welcome the approach of "Friedreich's Ataxia Awareness Day" on May 20th. The third Saturday of May is recognized in Congressional and state proclamations and through various events around the country as "Friedreich's Ataxia Awareness Day." Friedreich's ataxia is a fatal, hereditary, degenerative, neurological disease that typically first becomes noticeable when children are in elementary school.
An extraordinary explosion of research insights has followed the identification of the Friedreich's ataxia gene in 1996. Now, only ten years later, there is increasing conviction that treatments will soon be developed for Friedreich's ataxia and that the resulting insights will be broadly applicable across a wide range of neurological disorders such as Parkinson's, Huntington's, Alzheimer's, ALS and a number of rarer diseases as well.
This growing conviction and the accompanying excitement are based on the fact that this year there are plans for five clinical trials of drugs that show tremendous promise for treating Friedreich's ataxia for the first time. Friedreich's ataxia scientists and patient families alike are confident, in fact, that these clinical trials, and others that are to follow early next year, will actually result in the first treatments for this disease. Because such clinical trials are far more expensive than the basic research from which they grow, the "Awareness Day" events being planned across the country this year are more important than ever.
The disease often first becomes noticeable as a balance and coordination problem when children are in elementary school. Further loss of strength and coordination in all four extremities usually forces them into wheelchairs by their teens, while the disorder impairs vision, hearing and speech as it progresses. Additional symptoms frequently include severe heart disease, scoliosis, and diabetes. In the later stages of the disease, patients are usually completely incapacitated, often dying of cardiac failure. Average life expectancy extends only to early adulthood, although some people with less severe symptoms of Friedreich's ataxia live much longer. Friedreich's ataxia, although rare, is the most prevalent inherited ataxia, affecting about one in every 50,000 people in the United States.
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non profit, tax-exempt organization dedicated to the pursuit of educational, scientific and research activities leading to treatments for Friedreich's ataxia. FARA also serves as a patient advocacy group to educate the public, elected representatives, and other government officials regarding Friedreich's ataxia and the importance of funding biomedical research. FARA works closely with the National Institutes of Health and other organizations in the United States and elsewhere that are supportive of Friedreich's ataxia research. http://www.FAResearchAlliance.org
President, Friedreich’s Ataxia Research Alliance