News

In adults with Friedreich’s ataxia (FA), treatment with nomlabofusp may increase levels of frataxin protein to at least half of what’s typically seen in people who don’t have the disease, according to new analysis of clinical trial data. The analysis also predicted that use of the experimental therapy…

Key takeaways from the webinar include the complexity of the relationship between FA patients and caregivers, and the importance of practicing open communication and empathy and asking for help when necessary. Those are the key takeaways from a new webinar

Male mouse models of Friedreich’s ataxia (FA) in a study showed worse heart function than females, with weaker heart pumping and a larger left ventricle, the heart’s main pumping chamber. The differences are relevant as these mice are widely used for preclinical testing of new treatments for the disease. Researchers…

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…

PTC Therapeutics says it’s planning, this December, to ask the U.S. Food and Drug Administration (FDA) to approve its oral therapy vatiquinone — which has been tested in pediatric patients as well as adults — as a treatment for Friedreich’s ataxia (FA). “We look forward to submitting…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to PPL-001, Papillon Therapeutics’ investigational cell therapy for Friedreich’s ataxia (FA). This status is granted to potential medicines or therapy approaches meant to treat serious or life-threatening diseases affecting fewer than 200,000 people in…

A live, virtual roundtable on Nov. 6 at 6 p.m. EST will explore how people with Friedreich’s ataxia (FA) and caregivers can work together to deal with the everyday challenges of FA while living full lives. The free webinar, “Stronger Together: Patient and Caregiver Collaboration in FA,” is presented…

A recent mouse model of Friedreich’s ataxia (FA), called YG8-800, better mimics the disease’s symptoms and mechanisms in people than earlier models, making it a useful tool for testing treatments aiming to slow or stop disease progression, a study reports. Specially, YG8-800 mice carry a mutation that causes repeats…

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,”…