Skyclarys (omaveloxolone) improves heart function in Friedreich’s ataxia (FA) by reducing abnormal heartbeats and stabilizing heart rhythm, a study in a mouse disease model of severe cardiomyopathy concluded. Data also revealed sex-specific disturbance in the heart’s electrical activity and responses to Skyclarys treatment in these mice. The study,…
Throughout 2024, Friedreich’s Ataxia News brought readers detailed coverage of the latest clinical research related to Friedreich’s ataxia (FA), as well as updates on scientific breakthroughs and developments in treatment. Here are the top 10 most-read stories of last year, each with a brief description. We’re excited to continue…
Cardiomyopathy in people with Friedreich’s ataxia (FA) significantly associated with a shortening of chromosomal telomeres —  the “tips” of chromosomes — in immune cells, a study reported. Telomere length is known to decrease with age, and younger FA patients, up to around the mid-30s, had longer telomeres than…
Reduced muscle heath, physical inactivity, and more belly fat lowered sensitivity to insulin, a risk factor for diabetes, among adults with Friedreich’s ataxia (FA) who didn’t have diabetes, a small study finds. Further studies are needed to determine if muscle- or fat-focused interventions could delay FA-related diabetes, the…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to PPL-001, Papillon Therapeutics‘ cell therapy for Friedreich’s ataxia (FA). According to Papillon, PPL-001 uses a “unique multi-systemic approach to treat patients” with FA. The cell-based therapy “offers the potential to modify and reverse disease progression,”…
Widespread scar tissue in the lower left chamber of the heart — the left ventricle — correlated with genetic disease severity in people with Friedreich’s ataxia (FA), an imaging study found. Genetic disease severity refers to the amount of the frataxin protein derived from the least affected of a…
Larimar Therapeutics plans to launch a study by the end of the year that will test the pharmacological properties of its investigational therapy nomlabofusp, formerly CTI-1601, in children and adolescents, ages 2 to 17, with Friedreich’s ataxia (FA). It will begin with the enrollment of 12-15 adolescent…
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…
Researchers in a report described the first case of a Friedreich’s ataxia (FA) patient who developed photophobia, or an extreme sensitivity to light. A functional MRI scan of the 64-year-old woman showed abnormal brain activity in response to light stimulus compared with a scan from a non-FA patient with…
Voyager Therapeutics has selected a lead gene therapy candidate to advance into clinical trials with the aim of correcting the genetic defect that causes Friedreich’s ataxia (FA). With this decision, which was made with Neurocrine Biosciences, the companies expect to begin clinical trials in 2025. “The nomination…
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