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,”…
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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…
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…
Design Therapeutics is on track to start clinical trials next year of its new and improved experimental treatment for Friedreich’s ataxia (FA), called DT-216P2. “Leading our portfolio of potential first- or best-in-class therapies is DT-216P2 for FA, a serious neurodegenerative disease with a significant need for new therapies,…
Diazoxide, a medication used to treat high blood pressure, protected cells — isolated from a person with Friedreich’s ataxia (FA) — against cellular damage caused by oxidative stress, suggesting it could be repurposed to treat the disease. Because diazoxide can’t make it to the brain, which is affected by…
Increasing the levels of a protein known as TSPO in glial cells — which provide nutrients to nerve cells and help with their communication — extended survival and improved mobility in a fruit fly model of Friedreich’s ataxia (FA). These preclinical findings suggest that “TSPO might be a relevant therapeutic target for this…
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