News

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,…

In addition to three recruiting clinical studies that were announced in June, an additional trial is seeking participants with Friedreich’s ataxia (FA) to investigate how the disease affects the brain, spinal cord, and cognition. Listed in a monthly newsletter released by the Friedreich’s Ataxia Research Alliance (FARA),…

Liam Dougherty, who has Friedreich’s ataxia (FA), and Katelyn Leader, whose sister has an undiagnosed form of spinocerebellar ataxia, are creating a new website to connect people with all types of disabilities to consumer products that can help them in their daily lives. Day Undefined co-founders Dougherty,…

Reata Pharmaceuticals has been granted a preliminary meeting with the U.S. Food and Drug Administration (FDA) to discuss the submission of a new drug application (NDA) for omaveloxolone, an investigational treatment for Friedreich’s ataxia (FA). According to a company press release, the pre-NDA meeting is scheduled…