News

An experimental therapy may help increase the protein that’s missing or deficient in Friedreich’s ataxia (FA) in people with a rare mutation in the FXN gene, a new study suggests. The therapy targets a rare mutation, known as c.165+5G>C, which has been reported in only three patients to date. The…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to LX2006, a gene therapy for cardiomyopathy in Friedreich’s Ataxia (FA). The status means the Lexeo Therapeutics candidate may be eligible for fast track review and extra FDA guidance during the regulatory process, which could…

A newly awarded $300,000 research grant will fund a study on heart fibrosis, or the accumulation of scar tissue, in people with Friedreich’s ataxia (FA). The Muscular Dystrophy Association (MDA) and Friedreich’s Ataxia Research Alliance (FARA) awarded the grant to researchers at Weill Cornell Medical Center and Indiana…

The U.S. Food and Drug Administration (FDA) has provided Larimar Therapeutics with clear expectations for an application seeking accelerated approval of nomlabofusp, its potentially disease-modifying therapy for Friedreich’s ataxia (FA), according to the drug developer. Accelerated approval is a type of conditional approval based on early clinical…

Biogen has initiated dosing in a Phase 3 clinical trial, called BRAVE, that’s testing the oral therapy Skyclarys (omaveloxolone) among children with Friedreich’s ataxia (FA) ages 2 to 15. Skyclarys already is approved in the U.S. and the European Union, among other locations, for FA patients ages 16…

The volume of gray matter in the brain region known as the cerebellum helps predict motor and cognitive benefits for Friedreich’s ataxia patients after noninvasive neuromodulation, a therapy that delivers mild electric currents to brain regions, a study found. Gray matter contains a high concentration of nerve cell bodies…

A high dose of the Friedreich’s ataxia (FA) treatment Skyclarys (omaveloxolone), far above the approved dose to match blood levels when the therapy is taken with food, did not affect heart function in healthy individuals, a study confirms. The study, “Effect of a Supratherapeutic Dose…

A new gene editing technique disrupted the mutations that cause Friedreich’s ataxia (FA) in a mouse model, a study reports. A type of base editing, the technique swaps one genetic building block, or nucleotide, for another. These edits can interrupt long repetitions of three-letter DNA sequences (trinucleotide repeats, or…

The approved Friedreich’s ataxia (FA) drug Skyclarys (omaveloxolone) was found to be generally safe and well tolerated in people with FA in the real world, according to a new U.S. study that tested the medication’s safety over one year. Most patients remained on Skyclarys for more than 12 months…

Scientists have designed molecules to correct the effect of DNA expanded repeats that cause Friedreich’s ataxia (FA), according to a new study. Using patient-derived cells, experiments demonstrated that these molecules, known as anti-gene oligonucleotides, or A-GOs, markedly boosted the production of frataxin, which is the protein that’s deficient in…