News

It had been a few years since Sean Baumstark, who is living with Friedreich’s ataxia (FA), came up with the idea of a cruise for people who, like him, have a rare disease. It wasn’t easy, coordinating it all. And it wasn’t until everyone was finally aboard, meeting…

The 2026 Annual Ataxia Conference (AAC) will bring participants together next week for a three-day event of learning, connection, and fun-filled activities. The meeting, organized by the National Ataxia Foundation (NAF), will be held April 9-11 at the Rosen Centre Hotel in Orlando, Florida. Advance registration…

A brain imaging marker linked to increased activity of support cells in the brain may help detect early disease activity in children with Friedreich’s ataxia (FA), a study found. Researchers using an advanced MRI technique found that levels of the marker, myo-inositol, were elevated in regions involved in movement,…

Nearly half of people with Friedreich’s ataxia (FA) in the U.K. have insufficient vitamin D levels, although these levels were not associated with measures of disease severity, a new analysis shows. In addition, nearly 30% of patients had vitamin D levels in the deficiency range, meaning their levels were…

In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…

Friedreich’s ataxia (FA) is characterized by defects in how cells regulate iron, which seem to differ by tissue and over time, according to a study using a new mouse model designed to better reflect the human disease. Specifically, animals showed an early buildup of iron in the brain and…

In adults with Friedreich’s ataxia (FA), a brain communication pathway connecting the cerebellum to the motor cortex, which coordinates and controls movement, showed signs of tissue shrinkage, disrupted brain signaling, and reduced energy production on MRI scans. According to the imaging study, FA-related changes across the so-called dentato-thalamo-cortical…

Following feedback from the U.S. Food and Drug Administration (FDA), Larimar Therapeutics is on track to soon submit an application seeking accelerated approval — which gets a drug to patients but still requires additional testing — of nomlabofusp, its protein replacement therapy for Friedreich’s ataxia (FA). Larimar recently…

Abnormal buildup of iron in mitochondria, the powerhouses of cells, may contribute to heart damage in Friedreich’s ataxia, according to a new study done in a mouse model. Data also indicate that heart cells have reduced ability to recycle damaged mitochondria in Friedreich’s ataxia. Collectively, the findings shed new…

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…