News

An observational study in Europe is using a mobile health app to better understand how Friedreich’s ataxia (FA) affects patients’ health-related quality of life, as well as their economic and psychosocial well-being, in real time. Called PROFA, the study is taking place across six centers in Germany, Austria, and…

Treatment with interferon gamma (IFN-gamma), an immune signaling molecule, helped to protect against damage in a cellular model of Friedreich’s ataxia (FA), a study reports. Although IFN-gamma failed to show clear benefits in clinical trials, these new findings “could help in the design of better therapeutic schedules and shed…

In a deal worth more than $7 billion, Biogen is acquiring Reata Pharmaceuticals, the developers of Skyclarys (omaveloxolone), which earlier this year became the first approved treatment for Friedreich’s ataxia in the U.S. “This is a unique opportunity for Biogen to bolster our near-term…

The U.S. Food and Drug Administration (FDA) has greenlighted a request by Larimar Therapeutics to test a 50 mg dose of CTI-1601 on people with Friedreich’s ataxia (FA) and cleared the company to begin an open-label extension study of the treatment. “Gaining clearance to advance to a…

MRI scans detect large- and small-scale changes in various areas of the brain and brainstem in the early stages of Friedreich’s ataxia (FA) over time, a new study reports. Many of the changes occurred outside the cerebellum, a brain region primarily implicated in the motor problems associated with…

Inducing oxidative stress — a type of cellular damage — into nerve cells of mice caused symptoms similar to those observed in Friedreich’s ataxia (FA) patients, a study reported. Animal symptoms included a loss of motor coordination (ataxia), cardiac issues, nerve cell degeneration, and problems in the workings of…

People with Friedreich’s ataxia (FA), regardless of age, have a thinner layer of nerve cells in the eye’s retina compared with unaffected individuals, a study involving almost 200 patients found. Such thinning, which correlated with neurological disease severity and vision loss, continued to increase over time. Researchers believe retinal…

In-depth monitoring and prompt treatment enabled a man with Friedreich’s ataxia (FA) to successfully undergo a thyroidectomy — thyroid gland removal surgery — despite active symptoms of a life-threatening “thyroid storm” during the procedure, according a recent case report. A thyroid storm is a medical emergency that arises when…

A first group of patients has been treated with LX2006, an investigational gene therapy for heart disease in people with Friedreich’s ataxia (FA), in a Phase 1/2 clinical trial and dosing has begun in a second group. LX2006 has been well tolerated and not associated with unexpected…

Continuous exposure to low levels of oxygen, or hypoxia, prevented or rapidly reversed motor symptoms in a mouse model of Friedreich’s ataxia, a study suggested. While a continuous mild hypoxia regimen also slowed the onset of motor symptoms, it didn’t reverse motor impairment. Intermittent hypoxia had a detrimental effect…