Three mouse models of Friedreich’s ataxia (FA) do not accurately reflect FA-associated cardiomyopathy — a serious heart complication — as seen in patients, underscoring the difficulties in modeling this complication, a study reports. Across these different models, only one exhibited gene activity changes in the heart consistent with cardiomyopathy…
Sparking conversations about ataxia — a group of disorders that include Friedreich’s ataxia believed to affect 26 of every 100,000 children globally — will be the chief aim of this year’s annual International Ataxia Awareness Day (IAAD), set for Sept. 25. The 23-year-old event is organized by the…
People with Friedreich’s ataxia who were treated with Skyclarys (omaveloxolone) in the MOXIe trial and its extension for three years had more than 50% slower disease progression compared with patients in a natural history study, according to a new analysis. “The present results show a meaningful slowing of…
Worse ataxia at an initial assessment predicted poor outcomes after four years in people with Friedreich’s ataxia (FA), according to a natural history study. The speed of disease progression was different, fastest in those in the earliest disease stages and gradually slowing as it advanced. Being younger at the…
Researchers have developed a new mouse model of Friedreich’s ataxia (FA) that captures coordination and muscle strength impairment seen in patients, a study reports. The model was created by worsening symptoms of a standard mouse model by further suppressing production of the frataxin protein, which is deficient in FA…
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…
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