News

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…

SGT-212, a gene therapy candidate for Friedreich’s ataxia (FA) that’s currently in early clinical testing, has been granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA). The FDA awards this status to experimental treatments that are designed to address rare diseases that primarily affect children,…

In Friedreich’s ataxia (FA), a subset of patients develops severe thickening of the heart muscle as the heart shifts to using glucose (sugar) as a fast but less effective energy source. This switch is linked to poorer heart function and early signs of damage, a study suggests. These patients…

An experimental gene therapy injected directly into the eye preserved the structure and function of the retina — the light-sensitive tissue at the back of the eye — in mouse models of Friedreich’s ataxia (FA), according to a proof-of-concept study. These data support “continued efforts toward the development of…