News

The U.S. Food and Drug Administration (FDA) has granted its fast track designation to Reata Pharmaceuticals’ oral candidate omaveloxolone for Friedreich’s ataxia (FA). Fast track status is designed to accelerate the development of investigational therapies that address unmet medical needs in serious or life-threatening conditions. It makes Reata eligible…

People who have interruptions in a specific part of their GAA repeats in the FXN gene experience the onset of Friedreich’s Ataxia (FA) about nine years later than those without such breaks, a study found. These findings highlight the importance of GAA repeat interruptions on the course of FA,…

Design Therapeutics announced that it remains on track to launch the first clinical trial of its lead experimental candidate — gene-targeted chimera (GeneTAC) — for the treatment of Friedreich’s ataxia (FA) for the first half of 2022. The planned Phase 1 trial will test the therapy in patients,…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…

This year’s rideATAXIA Dallas, a cycling event that raises money to support research into treatments for Friedreich’s ataxia, will take place Nov. 6. The event will feature in-person rides at UNT Discovery Park in Denton, Texas. Four route options are available: three, six, 27, and 50 miles. Registration…

A Phase 2a trial assessing Stealth BioTherapeutics‘ lead investigational product elamipretide in people with Friedreich’s ataxia (FA) is expected to initiate by year’s end. After feedback from the U.S. Food and Drug Administration, the company amended the trial’s design. It now aims to recruit 16 FA patients with…

Reata Pharmaceuticals is planning to ask the U.S. Food and Drug Administration (FDA) to approve omaveloxolone to treat people with Friedreich’s ataxia (FA) early in 2022, following the completion of a preliminary meeting with the agency. “Omaveloxolone could be the first drug approved for the treatment of Friedreich’s…

The third United Against Ataxia Hill Day will take place virtually Sept. 29, an event set aside to heighten awareness and support legislation that affects the ataxia community, including those with Friedreich’s ataxia (FA). All U.S. residents are invited to participate in the day-long advocacy event, which is a…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

Supporters globally are gearing up for International Ataxia Awareness Day, observed annually on Sept. 25 to raise the profile of a group of progressive neurological disorders that includes Friedreich’s ataxia (FA). Patients, caregivers, and supporters will share stories on social media, advocate on behalf of the ataxia community,…