News

The U.S. Food and Drug Administration (FDA) will maintain a hold on the clinical development of CTI-1601, Larimar Therapeutics’ experimental treatment for Friedreich’s ataxia (FA), which was originally paused for safety concerns and a request for more data. Larimar is now analyzing data from…

Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…

The National Ataxia Foundation (NAF) is accepting applications for funding of research projects on ataxia — a lack of motor coordination that also affects people with Friedreich’s ataxia — from minority students pursuing doctoral studies. Eligible students include U.S. citizens or permanent residents from underrepresented minorities who are…

Reata Pharmaceuticals has initiated a rolling submission of a new drug application seeking U.S. approval of its experimental oral therapy omaveloxolone for people with Friedreich’s ataxia (FA). In a rolling submission, companies can submit individual sections of the application for review by the U.S. Food and Drug Administration (FDA)…

A researcher in Australia has received $250,000 from the Friedreich’s Ataxia Research Alliance (FARA) to use stem cells to understand the mechanisms behind the progressive loss of proprioception — the perception of the body’s position and movement — in people with Friedreich’s ataxia (FA). The two-year grant was…

Clinical trials are more attractive to people with ataxias — including those with Friedreich’s ataxia (FA) — when they target specific symptoms, such as walking and balance issues; provide potential benefits to patients and others; and cover costs of travel and accommodation, according to a recent study. These were…

Creatine chemical exchange saturation transfer or CrCEST, a metabolic imaging tool, may be a valuable technique for detecting mitochondrial function across muscles in people with Friedreich’s ataxia (FA), a new study found. To date, CrCEST has not been used to assess mitochondrial capacity in people with FA. “CrCEST MRI…

Exicure is drawing to a close its XCUR-FXN program for the treatment of Friedreich’s ataxia for an unknown period of time, the company announced. The decision comes after an internal investigation concluded that data from the program’s preclinical phase would not support steps into a clinical trial, planned…

Shandra Trantham, 24, was a naturally inquisitive child and loved science. So when she was diagnosed with the rare neuromuscular disease Friedreich’s ataxia (FA) around age 9, she set out to learn as much as she could. “When I had this diagnosis, and I read online that there was…

Jacob Thompson began to hear an incessant ticking noise emanating from the engine of his 2002 Jeep Grand Cherokee. He played music to drown it. In doing so, he ignored a looming problem that ultimately put his car out of commission. That’s the metaphor Thompson uses when he describes his…