News

Skyclarys (omaveloxolone) improves heart function in Friedreich’s ataxia (FA) by reducing abnormal heartbeats and stabilizing heart rhythm, a study in a mouse disease model of severe cardiomyopathy concluded. Data also revealed sex-specific disturbance in the heart’s electrical activity and responses to Skyclarys treatment in these mice. The study,…

People with Friedreich’s ataxia (FA) were found in a new study to experience more mental health symptoms — particularly decreased motivation and greater anxiety and depression — than individuals without the rare disease, who served as controls. The results of this survey study, conducted in the Czech Republic, also…

Two microRNAs (miRNAs) — tiny molecules that control the activity of genes — are associated with structural brain and heart damage in Friedreich’s ataxia (FA) patients, according to a study from Brazil. FA patients were found to have higher levels of one miRNA, and lower levels of…

Adolescents with Friedreich’s ataxia (FA) are now being dosed with nomlabofusp as part of a placebo-controlled Phase 1 clinical study that’s testing Larimar Therapeutics‘ subcutaneous, or under-the-skin, injection therapy. Data from the Phase 1 study, and from other clinical testing of nomlabofusp, are expected by the middle…

Errors during repair when DNA gets damaged may give rise to the mutations that cause Friedreich’s ataxia, a study revealed. The study, “Recurrent DNA nicks drive massive expansions of (GAA)n repeats,” was published in PNAS. A cell’s DNA stores the instructions to make the proteins and…

The U.S. Food and Drug Administration (FDA) has given Solid Biosciences the go-ahead to start clinical testing of SGT-212, the company’s novel gene therapy candidate for Friedreich’s ataxia (FA). According to Solid, SGT-212 is the only experimental gene therapy of its kind to target “both neurologic and…

Throughout 2024, Friedreich’s Ataxia News brought readers detailed coverage of the latest clinical research related to Friedreich’s ataxia (FA), as well as updates on scientific breakthroughs and developments in treatment. Here are the top 10 most-read stories of last year, each with a brief description. We’re excited to continue…

Note: This story was updated Dec. 19, 2024, to clarify that the BOLD study does include measures of efficacy. Since its 2023 launch as the first and — to date — only approved treatment for Friedreich’s ataxia in the U.S., Skyclarys (omaveloxolone) has benefited most patients by either…

A genetic analysis of connective tissue cells from a woman with late-onset Friedreich’s ataxia (FA) and her younger sister, who did not have the disease, found differences in the activity levels of almost 400 genes in the affected sister, according to a recent study. Genes with the most altered…

Data from wearable sensors that are designed to collect real-world information about a person’s ability to function in daily life correlated well with standard clinical assessments of disease severity among people with Friedreich’s ataxia (FA), a study finds. These types of remote monitoring tools could help to better track…