News

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…

Voyager Therapeutics expects to begin clinical testing of its experimental gene therapy for Friedreich’s ataxia (FA) in 2026. The company last year announced the selection of its FA gene therapy candidate, which is being developed in collaboration with Neurocrine Biosciences. At the time, Voyager had said…

Children with Friedreich’s ataxia are often underweight and short for their age, while adult patients are frequently overweight, so regular nutrition checks are important for maintaining healthy weight, a study from Europe suggested. The study, “Longitudinal analysis of anthropometric measures over 5 years in patients with Friedreich ataxia in…

Fundraisers, webinars, and social media campaigns are set for next month to help bring attention on Friedreich’s ataxia (FA), and provide support to those living with the disease. Leading those efforts is the Friedreich’s Ataxia Research Alliance (FARA), which is dedicated to raising funds and aligning efforts for…