News

A group of brain diseases known as sporadic late-onset acute cerebellar ataxias have been attributed to lots of causes, so an American research team decided to study 80 patients to try to find patterns. They identified some patterns, but their two-year study’s findings basically backed up scientists’ long-held contention that there are…

Molecules released by certain stem cells may promote an increase of frataxin protein and rescue cellular function, researchers have shown in a cell model of Friedreich’s ataxia. The study, “Mesenchymal Stem Cell-Derived Factors Restore Function to Human Frataxin-Deficient Cells,” was published in the journal The Cerebellum. Friedreich’s ataxia is…

Researchers working in Friedreich’s ataxia may submit their project proposals for funding by two grants awarded by the Friedreich’s Ataxia Research Alliance (FARA), the Kyle Bryant Translational Research Award and the Bronya J. Keats International Research Collaboration Award. To apply researchers need to submit a letter of intent…

Patients with Friedreich’s ataxia (FA) and caregivers may benefit from participating in the upcoming Patient-Focused Drug Development meeting by the U.S. Food and Drug Administration (FDA). The meeting will be held at the College Park Marriott and Conference Center, Hyattsville, Maryland, on June 2 (8 a.m.-12:30 p.m.). After the meeting, there…

Low levels of frataxin protein, the hallmark of Friedreich’s ataxia, do not seem to impact the function of red blood cells, particularly the formation of hemoglobin’s core molecule, heme, a study suggests. The finding casts doubt on some researchers’ supposition that iron-metabolism problems play a role in Friedreich’s ataxia. It…

Gait speed might be used as an objective measure to assess disease progression in adults with Friedreich’s ataxia who are still able to walk, according to a small study which suggested that the measure is more sensitive in detecting neurological changes in the short term. Because current tools for measuring…

A type of compound called HDAC inhibitors show promise as therapy for Friedreich’s ataxia, according to two researchers at The Scripps Research Institute in California. However, current drug candidates need to be improved to be suitable for use in a chronic condition such as Friedreich’s ataxia. In the study,…

Friedreich’s ataxia (FRDA) stems from abnormal DNA code expansions in the Frataxin (FXN) gene that collectively reduce the expression of the FXN protein. A new study indicates that small RNA and DNA molecules can interact with FXN repeats to block the formation of the abnormal DNA 3D structures that could be…

Voager Therapeutics has announced that its Friedreich’s ataxia program, VY-FXN01, is taking steps to initiate preclinical tests of its safety and effectiveness, the company expects to achieve in a two-year period the needed requirements to proceed to treatment testing in Friedreich’s ataxia patients. “Voyager’s exceptional performance and accomplishments during…

A Turkish study has classified the genetic and observable characteristics of Friedreich’s ataxia (FA) and spinocerebellar ataxia (SCA) in patients diagnosed with hereditary cerebellar ataxia. The study, “Determination of Genotypic and Phenotypic Characteristics of Friedreich’s Ataxia and Autosomal Dominant Spinocerebellar Ataxia Types 1, 2, 3, and 6”, appeared in the…