News

One of the most encouraging developments in ataxia research these days is a surge in therapies advancing to the clinical trial stage. This has put a premium on trial design, which means that many of those going to the Second International Ataxia Research Conference (IARC) in Pisa, Italy, set for…

Dozens of scientists from universities and pharmaceutical companies will come together at the 2nd International Ataxia Research Conference in Pisa, Italy, Sept. 27-30 to discuss new strategies for addressing the underlying mechanisms of ataxias. The conference will focus in particular on inherited ataxias, or those with a genetic cause. Since…

Missing 184 class periods in a year due to fatigue. Having someone tell the school principal you’re drunk because of your walking problems. Being belittled because of your slurred speech or hearing problems. Those were some of the situations that Friedreich’s ataxia patients told U.S. Food and Drug Administration officials…

Editor’s Note: The 2017 symposium had to be canceled because of Hurricane Irma, but the Energy Ball event was held as planned. The Friedreich’s Ataxia Research Alliance teams up with the University of South Florida next week on a patient-oriented symposium spotlighting the latest developments in the quest to cure…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Jotrol, a potential new therapy for Friedreich’s ataxia (FA), says the medication’s developer, Jupiter Orphan Therapeutics. The FDA decision “positions our Jotrol product as a potential treatment for FA … patients around the world,” Jupiter CEO…

Research into how abnormalities in the large area of the brain are part of Friedreich’s Ataxia disease processes will advance understanding and improve the chances of finding new treatments, researchers from Australia’s Monash University contend in a review. But research needs to focus on larger, multi-modal studies that will allow both…

The first International Ataxia Research Conference, in 2015, was a milestone in the quest to find cures for the world’s 40 ataxias, diseases whose hallmark is the loss of muscular coordination. Held in Windsor, England, it was the largest global convention yet to bring together patients, doctors, patient advocacy groups, researchers, pharmaceutical companies, regulators…

Researchers at the Children’s Hospital of Philadelphia (CHOP) have linked the presence of diabetes in Friedreich’s ataxia (FA) patients to more severe symptoms and a reduced capacity to manage daily living tasks. These insights suggest that identifying patients with diabetes and more actively managing their disease might help improve their health…

Chondrial Therapeutics‘ CTI-1601, an investigative therapy for Friedreich’s ataxia (FA), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). “We are very pleased to receive orphan drug designation for CTI-1601. Our team is working to complete all Investigational New Drug (IND) enabling studies so that…

The U.S. Food and Drug Administration (FDA) agreed that Reata Pharmaceuticals can use the modified Friedreich’s Ataxia Rating Scale (mFARS) as a primary endpoint for the second part of its possibly pivotal Phase 2 clinical trial assessing omaveloxolone in treating Friedreich’s ataxia, the company announced. This decision followed Reata’s request that the FDA confirm its earlier guidance that,…