News

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…

Larimar Therapeutics has begun the process of asking the U.S. Food and Drug Administration (FDA) to grant accelerated approval to nomlabofusp, its protein replacement therapy for Friedreich’s ataxia (FA). Typically, drug developers seeking a therapy’s approval need to finish the entire application before submitting it to the FDA.

Use of the experimental gene therapy LX2006 is generally safe and appears to improve or stabilize markers of heart health among people with Friedreich’s ataxia (FA). That’s according to newly published data from two early clinical trials that tested the one-time infusion therapy in a small number of…

Two microRNAs (miRNAs) — molecules that help regulate gene activity — may serve as biomarkers of cardiomyopathy in people with Friedreich’s ataxia (FA), according to results of a recent study. Combining the levels of these two miRNAs, called miR-323a-3p and miR-625-3p, into a single predictive model showed strong ability…

A Phase 2 clinical trial testing Lexeo Therapeutics’ gene therapy LX2006 in people with Friedreich’s ataxia (FA) and the heart condition cardiomyopathy expects to enroll its first participant by the end of the month, the company said. The SUNRISE-FA 2 study is a pivotal trial, with data…

Biogen’s Skyclarys (omaveloxolone) is one step closer to being covered by Quebec public healthcare systems for people with Friedreich’s ataxia (FA), ages 16 and older, with the province’s Institut national d’excellence en santé et en services sociaux’s (INESSS) re-evaluation of the therapy after initially declining to recommend it.