Marisa Wexler, MS,  senior science writer—

A new mouse model called YG8-800, which accurately captures key features of Friedreich’s ataxia such as neurological dysfunction and heart disease, could be a useful tool for studying genetic therapies for the disease. That’s according to the study, “A promising mouse model for Friedreich Ataxia progressing…

The Friedreich’s ataxia community wants to get people talking about ataxia — a group of disorders that affect co-ordination, balance, and speech — for this year’s International Ataxia Awareness Day (IAAD), which takes place on Sept. 25. “A great way to spread awareness is to encourage people to…

Skin cells of people with Friedreich’s ataxia (FA) show dysregulated levels of fat molecules, including abnormal levels of a class of fats called ceramides that are important for maintaining cellular membranes, a new study shows. Moreover, the researchers noted that impairments in fat molecules known as lipids “predicted the…

Note: This story was updated on Aug. 10, 2022, to add the U.S. Food and Drug Administration has extended its review of the omaveloxolone application through the end of February. Following a meeting with the agency, Reata Pharmaceuticals has submitted new data to the U.S. Food and Drug Administration…

Mutations that cause Friedreich’s ataxia lead to abnormalities in how nerve fibers grow and reduce their ability to repair themselves, according to a study done in mouse cells. These differences may contribute to nerve cell degeneration that is characteristic of the disease, researchers say. The study, “Frataxin…

Astrocytes, a type of support cell in the brain, showed metabolic defects and unusual pro-inflammatory activity that likely contributes to neuronal dysfunction in a cell model of Friedreich’s ataxia (FA), according to a new study. Results also indicate that treatment with a molecule called smoothened agonist (SAG) can normalize…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

The U.S. Department of Defense (DoD) is offering grant awards to researchers working to improve care for Friedreich’s ataxia. The DoD’s Congressionally Directed Medical Research Programs (CDMRP) is a funding organization whose goal is to further the development of new approaches to address specific areas of biomedical research as…

Targeting the end regions of FXN mRNA – the intermediary molecule used to make frataxin – may allow cells to make more of this protein, whose absence causes Friedreich’s ataxia, a new study shows. “We demonstrated a novel approach to increase intracellular levels of FXN mRNA and protein based…

A team of scientists at Koc University in Turkey has created three new cell models of Friedreich’s ataxia (FA) derived from patients with repeat expansions in their FXN gene. The models “will facilitate studies to understand molecular mechanisms related to FRDA [Friedreich’s ataxia] pathology [disease processes] as well as therapeutic…