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…
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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,”…
A newly awarded research grant from the Friedreich’s Ataxia Research Alliance (FARA) and FARA Ireland will help Stealth Biotherapeutics to evaluate the potential of its novel therapy SBT-589 for treating nerve damage in Friedreich’s ataxia (FA). The grant, the Kyle Bryant Translational Research Award — named for the…
Boosting the breakdown of fat promoted higher levels of frataxin, the deficiency of which is a hallmark of Friedreich’s ataxia, and reduced the risk of diabetes in a mouse model of the disease. The study, “Interplay of…
A brain pathway that connects the cerebellum to the motor cortex, helping coordinate and control movement by sending important signals between these two areas, is damaged in Friedreich’s ataxia (FA). In an imaging study, researchers observed that the dentato-thalamo-cortical tract (DTT) is most damaged near the cerebellum at the…
People with Friedreich’s ataxia often show abnormalities in their eye movements and looking for characteristic changes there may help diagnose the disease and track its progression, a review study finds. “Quantitative oculomotor [eye movement] testing in [Friedreich’s ataxia] may facilitate early diagnosis and provide value in monitoring disease…
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…
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