Hot topics of the Nov. 1 webinar included the challenges of living with Friedreich's ataxia, strategies for success, and the recent "game-changing" therapy approval. Read more about the highlights here.
Therapies that aid mitochondria, cellular organelles responsible for producing most of the energy needed by cells, may help with physical function and heart health in people with Friedreich’s ataxia (FA), according to a review of published studies. Findings appear to support the benefits of Skyclarys (omaveloxolone), the only…
Analyzing patients’ voices using machine learning may help to identify Friedreich’s ataxia (FA) and differentiate it from other neurological disorders, a new study shows. “The implications of this approach are substantial and provide new opportunities for healthcare, particularly for remote and rural areas where access to health providers might…
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…
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…
Measuring abnormalities in the neurological circuits the brain uses to make sense of sound could be a useful way to track the severity of Friedreich’s ataxia in people with the disease as well as in laboratory models, a new study reports. The study, “Auditory neuropathy in…
Fredrich’s ataxia (FA) impairs the regulation of hundreds of genes in muscle cells, resulting in reduced activity in those responsible for mitochondrial function and increased activity of repressor genes, a study reports. Addressing both aspects of this genetic abnormality “double hit” may be necessary to achieve the best therapeutic…
A new assay to measure levels of the frataxin protein in blood could be used to assess the effectiveness of treatments in early clinical trials. The researchers showed that measuring frataxin with this method, which uses pig blood as part of the standardization procedure, could distinguish between people with or…
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