Margarida Maia, PhD, science writer —

People with Friedreich’s ataxia (FA) who also have diabetes, a condition that causes their blood glucose (sugar) level to become too high, are more likely than those who don’t have diabetes to have worse outcomes from COVID-19, according to a single-center study. Having diabetes increased both the chance of…

A four-week Phase 2 clinical trial that’ll be testing CTI-1601, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), is now enrolling in the U.S., the company announced in a press release. Patient recruitment began shortly after the U.S. Food and Drug Administration (FDA) lifted its full hold…

The Phase 1 clinical program of DT-216, Design Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), remains on track, with initial data expected by year’s end. The program includes a single-ascending dose Phase 1a trial (NCT05285540) and a multiple-ascending dose Phase 1b study (NCT05573698), both testing the…

Some proteins in the cerebrospinal fluid (CSF) — the fluid that flows around the brain and spinal cord — are present in unusual amounts in patients with Friedreich’s ataxia, and may be candidate biomarkers for diagnosis, a small study suggests. Researchers found that the proteins are involved in how…

Some molecules involved in metabolism — all daily operations run by cells — are found in different amounts in patients with Friedreich’s ataxia (FA) versus healthy individuals and may be used as both diagnostic biomarkers and therapeutic targets, a small study suggests. The study, “Metabolomics analysis reveals dysregulation…

DNA repeats in the gene that codes for frataxin — a protein that’s not made enough in Friedreich’s ataxia — act as a molecular “stop sign” that results in no working protein being made, a study in lab-grown cells found. Researchers were able to offset the “stop sign” and…

Children and adults with Friedreich’s ataxia (FA) have lower lean body mass — simply put, the weight of everything in the body except fat — than healthy individuals of about the same age, a study found. Using mice, researchers showed that this loss of lean body mass occurred…

The U.S. Food and Drug Administration (FDA) has granted priority review to Reata Pharmaceuticals’ omaveloxolone, an investigational once-daily oral therapy for Friedreich’s ataxia. If approved, omaveloxolone would become the first therapy available for the treatment of the disease in the U.S. The decision comes following the company rounding…

A researcher in Australia has received $250,000 from the Friedreich’s Ataxia Research Alliance (FARA) to use stem cells to understand the mechanisms behind the progressive loss of proprioception — the perception of the body’s position and movement — in people with Friedreich’s ataxia (FA). The two-year grant was…

Exicure is drawing to a close its XCUR-FXN program for the treatment of Friedreich’s ataxia for an unknown period of time, the company announced. The decision comes after an internal investigation concluded that data from the program’s preclinical phase would not support steps into a clinical trial, planned…