News

When you’re living with Friedreich’s ataxia (FA), the period following a diagnosis can be filled with uncertainty, including when you’re preparing for your first visit with a neurologist. At the 2026 Annual Ataxia Conference (AAC) in Orlando, Florida, a recent session provided guidance on navigating an appointment with…

Scientists have created and validated stem cell lines derived from an adult with Friedreich’s ataxia (FA) carrying an ultra-rare disease-causing mutation, a study reports. Because patient-derived stem cells can be transformed into any cell type — including muscle and nerve cells, which are most affected in FA, a rare…

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…