News

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…

Larimar Therapeutics has received a patent relative to CTI-1601, its experimental and potentially disease-modifying treatment for Friedreich’s ataxia (FA), the company announced. The patent, No. 11,459,363 and titled “Materials and Methods for Treating Friedreich’s Ataxia,” provides protection for composition of matter relative to CTI-1601 until at least July 2040.

In a reversal, the U.S. Food and Drug Administration (FDA) has informed Reata Pharmaceuticals that it will not hold an advisory committee meeting to discuss the company’s application for omaveloxolone, its oral treatment candidate for Friedreich’s ataxia. The FDA’s reversal means that patients and the FA community will…

A mutation in the gene SIRT6 is associated with less severe neurological symptoms and visual impairment among people with Friedreich’s ataxia, a new study reports. “We have identified a [mutation] in SIRT6 as a potential modifier of [Friedreich’s ataxia] neurological features,” researchers wrote. “The less common CT genotype…

A new mouse model called YG8-800, which accurately captures key features of Friedreich’s ataxia such as neurological dysfunction and heart disease, could be a useful tool for studying genetic therapies for the disease. That’s according to the study, “A promising mouse model for Friedreich Ataxia progressing…

Higher than normal activation of the mTOR and AKT signaling pathways — both key to proper cardiovascular function — may underlie the heart disease called cardiomyopathy that is common in people with Friedreich’s ataxia (FA), according to a new study using a mouse model of FA. When the mice…

The Friedreich’s ataxia community wants to get people talking about ataxia — a group of disorders that affect co-ordination, balance, and speech — for this year’s International Ataxia Awareness Day (IAAD), which takes place on Sept. 25. “A great way to spread awareness is to encourage people to…

A substantial knowledge gap still exists about the prevalence of Friedreich’s ataxia (FA) around the world, as well as its impact on the quality of life of patients and caregivers, and care costs, a review study has found. Nevertheless, the limited available data point to physical impairment as the…

Repeat use of G-CSF stem cell therapy, an approved medicine, was safe in adults with Friedreich’s ataxia and associated with significant elevations in frataxin protein and disease-related biomarkers, a pilot study in the U.K. reported. Because interventions that increase frataxin and reverse the harmful effects of the protein’s deficiency are…

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…