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…
Three researchers in the U.S. have each been awarded a grant from the Friedreich’s Ataxia Research Alliance (FARA) to explore new strategies for treating Friedreich’s ataxia (FA), the nonprofit announced on its scientific news page. The newest FARA grant recipients are three professors from across the U.S.
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…
MRI imaging indicates that Friedreich’s ataxia (FA) patients have impairments in proprioception — sensory processing related to body movement and location — relative to healthy adults, but these changes do not contribute to overall disease severity, according to recent research. Brain responses to tactile stimulation (light touch) were similar…
Researchers are conducting a Phase 2 clinical trial to evaluate the safety, efficacy, and molecular effects of dimethyl fumarate (DMF), an approved treatment for multiple sclerosis (MS) and psoriasis, in people with Friedreich’s ataxia (FA). Based on both preclinical research and clinical studies in MS patients, it…
Supplementation with butyrate, a short-chain fatty acid produced in the gut by good bacteria, reversed many diabetes-like metabolic defects found in fat tissue from a mouse model of Friedreich’s ataxia, a study reported. In addition to its effects on metabolism, butyrate lowered signs of ongoing inflammation observed in FA…
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…
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