News

Levels of iron in the blood, along with iron stored in the liver and spleen, are significantly lower in adults with Friedreich’s ataxia (FA) relative to healthy adults, and the more severe the FA-causing mutation, the lower the iron in blood and organs, a study shows. The findings highlight…

A low dose of calcitriol, an activated form of vitamin D used to help manage hypoparathyroidism and other disorders, led to statistically significant increases in frataxin protein levels among people with Friedreich’s ataxia in a small clinical trial conducted in Spain. Although patients in the yearlong study did…

Thickening of the heart muscle, although rare, may be the first manifestation of Friedreich’s ataxia (FA) in children, according to a new study. These findings suggest genetic testing to confirm the FA diagnosis should be considered in children with unexplained hypertrophic cardiomyopathy, when the walls of the left…

The U.S. Food and Drug Administration (FDA) has selected nomlabofusp, an investigational treatment for Friedreich’s ataxia (FA), to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. Launched by the FDA in September 2023, the milestone-driven program seeks to accelerate the development of…

The U.S. Food and Drug Administration (FDA) has removed the partial hold on the clinical development of nomlabofusp, an experimental treatment for Friedreich’s ataxia (FA). The decision followed a review of data from a Phase 2 dose exploration trial (NCT05579691), which showed the treatment was well tolerated…

Thanks to a philanthropic donation of up to £650,000 (about $825,000), the Oxford-Harrington Rare Disease Centre (OHC) is offering two new grant programs to support the development of treatments for Friedreich’s ataxia (FA). The new awards, dubbed The FA Alliance Catalyst Fund and The FA Alliance Innovation Fund,…

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…

An experimental compound called ATH434, currently being evaluated in clinical trials for a neurological disorder called multiple system atrophy, also may have the potential to treat Friedreich’s ataxia (FA). Data specifically indicate that ATH434 could help to reduce the toxic buildup of iron in cells that is characteristic…

May will be a busy month for patients, doctors, researchers, and advocates in the Friedreich’s ataxia (FA) community as they participate in FA Awareness Month, an initiative for sharing information, personal stories, and support for those living with the rare neuromuscular disease. Named after Nikolaus Friedreich, the German physician…

The U.S. Food and Drug Administration (FDA) has put Lexeo Therapeutics’ LX2006, a gene therapy candidate for heart disease in people with Friedreich’s ataxia (FA), on the fast track for development. The award of fast track status aims to expedite the development and regulatory review of investigational therapies…