Lindsey Shapiro, PhD,  science writer—

Treatment with vatiquinone significantly slowed declines in upright stability among children and young adults with Friedreich’s ataxia (FA) in a clinical trial, leading researchers to predict it could delay the loss of patients’ ability to walk (ambulation) by about nine months. While the MOVE-FA clinical trial failed…

Use of the investigational therapy CTI-1601, now known as nomlabofusp, was associated with dose-dependent increases in levels of frataxin — the protein whose lack causes Friedreich’s ataxia (FA) — in skin and buccal cells of adult patients in a clinical trial. Treatment tolerability also was reported in top-line…

Diabetes, heart rhythm problems, and neurological disability were found to be the clinical factors most likely to influence life expectancy in people with Friedreich’s ataxia (FA) in a recent European registry study. Scientists used the information to generate a model, which also included left ventricle dysfunction, that doctors 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…

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…

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…

Disruptions to the DNA replication process during cell division may cause the expansion of FXN mutations that underlie Friedreich’s ataxia (FA), according to a recent study. Researchers have developed a new system to study the growth of these mutations — called GAA repeats — in human cells. They found that…