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News & Press Releases

Here you will find the most recent organizational news from FARA, including information on events, and awareness and advocacy initiatives. To locate an article from a certain date, please use the archives on the right side of your screen.

New study to examine whether smoking cessation drug Varenicline may benefit adults with Friedreich’s ataxia

A drug approved for smoking cessation may hold promise for people suffering from Friedreich’s Ataxia (FA), an inherited disease that causes progressive damage to the neuromuscular system. A new clinical study, sponsored by the Friedreich’s Ataxia Research Alliance (FARA), will investigate whether varenicline (Chantix®) improves neurological symptoms, such as balance, coordination, and sensory perception, all of which are significantly impaired in patients with FA. Chantix® is approved by the U.S. F ood and Drug Administration to help cigarette smokers stop smoking. Currently, there is no effective treatment for FA.

The double blind, randomized, placebo-controlled pilot study will be led by principal investigator Dr.Theresa Zesiewicz, professor of neurology at the University of South Florida College of Medicine, and co-investigator Dr . David Lynch, associate professor of neurology and pediatrics at Children’s Hospital of Philadelphia. Dr. Zesiewicz noticed that the uncoordinated movements (ataxia) and balance problems of a patient with fragile X tremor /ataxia syndrome imp roved greatly after he started varenicline in an attempt to quit smoking. The symptoms worsened when the medication was discontinued. Dr. Zesiewicz found similar results when treating patients with other types of ataxia, and several of her case reports were published last year in medical journals.

Read More:New study to examine whether smoking cessation drug Varenicline may benefit adults with Friedreich’s ataxia

Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint

Data from second Phase III study expected in the first half of 2010
Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on orphan neuromuscular diseases, announced today that its US Phase III clinical trial evaluating Catena® for the treatment of Friedreich's Ataxia missed its primary endpoint as measured by the International Cooperative Ataxia Rating Scale (ICARS). The study also did not show statistical significance on the second neurological endpoint, the Friedreich's Ataxia Rating Scale (FARS). On both endpoints, the active treatment arms showed a consistent improvement over baseline and placebo, as seen in prior studies. However, due to a lower than expected effect size combined with the fact that patients on placebo improved unexpectedly, statistical significance could not be achieved in this study population. The safety results were consistent with published data suggesting that Catena® is safe and well tolerated at doses up to 2250 mg/day.

Read More: Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint

Research Progress Fuels Preparations for "Friedreich's Ataxia Awareness Day"

Washington, D.C. — Friedreich's ataxia patients, families, and their communities eagerly welcome the approach of "Friedreich's Ataxia Awareness Day" on May 16th. The third Saturday of May is recognized i n Congressional and state proclamations and various activities across the country as "Friedreich's Ataxia Awareness Day.” Friedreich’s ataxia (FA) is a degenerative, neuromuscular disease that gradually robs patients of their ability to walk, compromises speech, hearing, and vision, and often comes with complications of serious diabetes and he art disease.

Following the identification of the Friedreich’s ataxia gene in 1996, research scientists have learned a great deal about the disorder. We now know what defects in the gene cause the disease, what protein the gene is supposed to produce, what that protein is supposed to accomplish, and why a shortage of the protein results in the cell death that leads to the disease symptoms. With this knowledge, researchers are better equipped to focus treatment development at the causes of the disease with the aim to slow, stop and reverse the effects of Friedreich’s ataxia.

Read More: Research Progress Fuels Preparations for "Friedreich's Ataxia Awareness Day"

Ride Ataxia III - Portland

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Ataxia is a disorder that may affect the fingers, hands, arms, legs, body, speech, and eye movements. Ride Ataxia's Kyle Bryant knows all too well the embarrassment that this disorder can bring. When he was younger, people chalked-up Kyle's incoordination to clumsiness. When he was diagnosed with Friedrich's Ataxia, Kyle felt like his life was over as there is no known cure. However, he discovered strenuous exercise helps alleviate his symptoms. Kyle started Ride Ataxia to help fund research and understanding of this complicated disorder. The Ride Ataxia Cycling Team has traveled 3,100 miles in the past two years raising invaluable Ataxia awareness and bringing together $350,000 in research funds in collaboration with the National Ataxia Foundation and the Friedrich's Ataxia Research Alliance. For more information call 484-875-3015.

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Cyclist with Rare Neuromuscular Disorder to Arrive in Seattle March 19 Completing 4 Day Cycling Journey

Seattle—Kyle Bryant and a team of 65 cyclists will arrive at the Doubletree Hotel Seattle Airport Thursday, March 19 at 2pm completing a 200 mile cycling journey to draw attention and raise research funds for the rare neuromuscular disorder Friedreich’s ataxia with which Bryant and other teammates, including Seattle native Sam Bridgman, are affected. Bridgman is a senior at Nathan Hale High School, works for the Seattle Mariners and will be cycling this year with his family and three of his close friends.

Bryant stated, “In 2009 Ride Ataxia looks forward t o continued success raising awareness, collaboratively funding research, and em powering ataxians and others to seek their fitness goals and have fun doing it.” The riders began their journey March 16 on the Oregon Health and Science University Campus in Port land. Bryant and the Ride Ataxia team will conclude the journey at the National Ataxia Foundation’s 52nd Annual Membership Meeting.

Read More: Cyclist with Rare Neuromuscular Disorder to Arrive in Seattle March 19 Completing 4 Day Cycling Journey

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