News

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…

Targeting enzymes that control how cells process fatty molecules called sphingolipids may offer a promising new treatment strategy for Friedreich’s ataxia (FA), a study in cells and mice has found. Researchers discovered that enzymes involved in sphingolipid metabolism were disrupted in several cellular models of the disease. Targeting two…

People whose Friedreich’s ataxia (FA) symptoms began in childhood or adolescence may face greater relationship-related challenges at more advanced disability stages than those whose symptoms started in adulthood, a study suggests. While both groups reported a similar overall number of adverse life events, relationship-related difficulties were more common among…

A new partnership aims to provide fast, consistent production of an experimental cell therapy for Friedreich’s ataxia. Papillon Therapeutics is teaming up with biological manufacturing company Cellares to automate the production of Papillon’s PPL-001, preparing the treatment for testing in clinical trials, and for commercial manufacturing…

The virtual care company Synapticure has launched a new program to offer remote neurological care for people with Friedreich’s ataxia and other types of ataxia. According to a press release from Synapticure, the new program can in some cases serve as a patient’s primary neurologist, or it…

Genetic mutations that cause Friedreich’s ataxia (FA) arise from a previously unrecognized category of FXN gene variants called a protomutation, according to a study. The protomutation was found exclusively in people of Eurasian descent and explains why the disease has always been limited to those populations. “These findings define…