Pfizer is seeking a European patent on a family of compounds that can treat Friedreich’s ataxia by increasing levels of the frataxin protein that is lacking in the disease. It noted in its patent application that Resverlogix has already developed a therapy called apabetalone (RVX-208) that is based on the compounds.
The Friedreich’s Ataxia Research Alliance (FARA) and the UPenn Center for Orphan Disease Research are sponsoring fundraising events May 20 to mark Friedreich’s Ataxia Awareness Day — an annual celebration to call attention to the rare, debilitating and often life-shortening degenerative neuromuscular disorder. Since its formation in 1998, FARA, a national, public,…
As a kid growing up in Seattle, Sam Bridgman lived and dreamed sports. An avid Seahawks fan, he loved football, baseball, hockey, soccer, skiing and boxing. In fact, world heavyweight champion Muhammad Ali was his idol, and “Impossible is Nothing” his mantra. But in 2006, Bridgman learned he had Friedreich’s…
The Friedreich’s Ataxia Research Alliance is holding a fun and easy photo challenge to raise global awareness of the disease. Photography buffs and amateurs can submit pictures of themselves with #CureFA signs to help the alliance mark Friedreich’s Ataxia Awareness Day on Saturday, May 20. You’ll need to submit the photos to the…
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…
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