News

Skyclarys (omaveloxolone) improved key measures of function and cellular health in nerve cells from the dorsal root ganglia, clusters of sensory nerve cells next to the spinal cord that are affected early in Friedreich’s ataxia (FA), according to a cell-based study. The researchers also found that combining low…

Scientists at the University of Oxford are embarking on five pioneering research projects — together backed by more than half a million dollars in funding — that aim to advance the discovery of new treatments for Friedreich’s ataxia (FA). The new projects are being funded by the Oxford-Harrington Rare…

Researchers have developed a new type of gene therapy that, according to the findings of a new study in animal models, may correct both nerve and heart problems in people with Friedreich’s ataxia (FA). The one-time treatment, administered via a spinal tap, was designed to simultaneously target the heart…

Sensors worn while walking at a natural pace can accurately distinguish people with Friedreich’s ataxia (FA) from those without the disease, according to a study. Data from these sensors also showed that FA patients with greater step-to-step variability and irregular trunk movements during walking had more severe disease. “Digital…

High-resolution nerve ultrasound revealed nerve enlargement, a sign of nerve changes, in the arms and legs of people with Friedreich’s ataxia (FA), a study in Germany shows. The study also found that some people showed nerve atrophy, or shrinkage, in the arms and legs. Together, the findings highlight that…

The first patient has been dosed in a small study testing SGT-212, a gene therapy candidate being developed by Solid Biosciences for people with Friedreich’s ataxia (FA). The dose-finding Phase 1b FALCON trial (NCT07180355) — a first-in-human study being conducted at two sites in the U.S. —…

The team at Friedreich’s Ataxia News spent 2025 bringing readers the latest updates on research, treatments, and clinical trials related to the rare inherited disease. From long-term real-world outcomes with approved therapies to promising steps toward gene therapy, this year delivered meaningful progress for the Friedreich’s ataxia (FA)…

Skyclarys (omaveloxolone) was well tolerated and associated with stable disease measures and reduced inflammation markers in adults with Friedreich’s ataxia (FA) over about six months, according to a real-world study in Italy. In particular, the treatment was associated with significantly lower levels of interleukin (IL)-6, a molecule involved…

An international team of scientists has modified a standardized neurological assessment so it can better measure the severity of non-movement symptoms in people with Friedreich’s ataxia (FA). The researchers found that the updated assessment closely matches established measures of FA severity. They said the findings highlight how often overlooked…

MRI scans analyzed with artificial intelligence (AI) to measure the size of the dentate nucleus — a deep brain region that tends to shrink in people with Friedreich’s ataxia — may help track how the disease progresses over time, according to a new study. “These volumetric changes were associated…