News

Using an experimental gene-editing approach, a team of U.S. scientists was able to successfully and precisely remove the genetic defect underlying most cases of Friedreich’s ataxia (FA) in several laboratory cell models. According to the researchers, the strategy achieved “precise editing” — as high as 98.2% — among connective…

People with Friedreich’s ataxia (FA) develop thickening of the heart muscle wall early on, followed by a gradual decline in pumping function that can take up to three decades to appear, according to a study that examined more than 25 years of echocardiogram records from nearly 600 FA patients.

A new documentary chronicling the story of two men with Friedreich’s ataxia (FA) as they undertake a seemingly impossible athletic endeavor — cycling across mountains, with a rare muscle disease — will premiere at a film festival in California later this month. The movie, “The Highest Road,” is…

Lexeo Therapeutics has acquired the private, clinical-stage company Mantle Therapeutics, gaining the rights to several experimental therapies Mantle had been developing for Friedreich’s ataxia (FA) — including one already in early clinical testing. In announcing the deal, which could be worth more than $21 million, Lexeo also heralded…

International Ataxia Awareness Day on Sept. 25 aims to bring people together worldwide to raise awareness of ataxia — a group of diseases that includes Friedreich’s ataxia (FA) — and the National Ataxia Foundation (NAF) is urging everyone to take part through advocacy, social media, virtual and in-person…

Replacing myeloid cells — a group of immune cells that includes microglia in the brain and spinal cord and macrophages in other tissues — with healthy, donor-derived cells improved neurological and heart abnormalities in a mouse model of Friedreich’s ataxia (FA), a U.S. study found. Microglia and macrophages can become…

Failure in a protein modification process needed to maintain the function of a brain receptor is a key early mechanism leading to Friedreich’s ataxia, a mouse study showed. The process, known as palmitoylation, links the loss of frataxin, the protein missing in people with FA, to nerve cell vulnerability…

A new gene therapy designed to restore frataxin protein to near-normal levels rather than produce excessive amounts improves movement, nerve signaling, and cell health in a mouse model of Friedreich’s ataxia (FA), a study reports. The treatment, called miniFXN7, was delivered in a single injection before symptoms began and used…

Sulforaphane, a compound found in broccoli and other vegetables, was shown in a laboratory study to improve nerve cell survival in Friedreich’s ataxia (FA) and to act on several biological processes implicated in the rare progressive disease. The scientists say the compound may be “a strong … drug candidate”…

Too little of the protein frataxin makes glial cells, which protect and nourish neurons, especially vulnerable to iron overload, while limiting how much iron is absorbed in the intestine may help extend survival and ease symptoms in Friedreich’s ataxia, according to a study that used flies to model the…