Lindsey Shapiro, PhD,  science writer—

Daily use of the experimental therapy nomlabofusp is leading to long-term clinical improvements and increases in frataxin levels for people with Friedreich’s ataxia (FA) relative to what would be expected in the absence of treatment. That’s according to new data from an ongoing open-label extension study (NCT06447025)…

Using MRI scans, a team of researchers identified several differences in the brain and spinal cord of people with Friedreich’s ataxia (FA) versus those without the rare genetic condition, which have the potential to serve as biomarkers of disease progression in clinical trials. The findings come from TRACK-FA…

The investigational gene therapy LX2006 was well tolerated and led to clinically meaningful improvements in cardiac biomarkers and functional measures in people with Friedreich’s ataxia (FA) and a type of heart disease called cardiomyopathy, the results from two clinical trials show. The findings informed the design of a…

CAP-004, Capsida Biotherapeutics’ experimental gene therapy for Friedreich’s ataxia (FA), safely delivered its genetic cargo to key disease-related tissues in nonhuman primates, new research shows. Specifically, that cargo was delivered to the heart, sensory nerves, and the central nervous system, or CNS, which comprises the brain and spinal…

Biogen’s Skyclarys (omaveloxolone) has been approved in Canada to treat people with Friedreich’s ataxia (FA) ages 16 and older, becoming the first disease-modifying therapy for FA to receive regulatory clearance in the country. The therapy was approved under Health Canada’s priority review process, which accelerates the review timeline…

The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…

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…

Inhibiting ferroptosis, a type of iron-dependent cell death, could be a promising therapeutic strategy for heart disease management in people with Friedreich’s ataxia, a study found. Through a series of experiments, scientists found that mice lacking a protein called SIRT3 in heart cells showed cellular properties similar to what’s…

Friedreich’s ataxia (FA) patients with less common types of disease-causing mutations in the FXN gene may have a distinct clinical presentation from patients with a more standard genetic profile, research suggests. A certain group of these mutations associated with higher production of frataxin — the protein lacking in FA — were…

A gene-editing therapy designed to correct the FXN gene defect that’s associated with Friedreich’s ataxia (FA) reversed several cellular features of the disease in patient-derived nerve cells, according to recent research. Scientists identified that dysfunction of a cellular compartment called the endoplasmic reticulum (ER) might play a key role in…