With backing from a petition that is now circulating online, the Friedrich’s Ataxia Research Alliance (FARA) will ask Reata Pharmaceuticals and the U.S. Food and Drug Administration to make omaveloxolone (RTA 408) available to people living with Friedrich’s ataxia (FA) — for whom no approved treatments currently exist.
Larimar Therapeutics completed dosing for its single-ascending dose (SAD) Phase 1 clinical trial of CTI-1601, a modified version of frataxin, in the treatment of Friedrich’s ataxia (FA), the company announced. A safety review committee cleared the trial to continue after evaluating preliminary blinded data from each of the…
The Friedreich’s Ataxia Research Alliance (FARA) has shared a list of three clinical studies and one global registry that are open and enrolling people with Friedreich’s ataxia (FA) in the search for more effective therapies. This rare disease is caused by abnormally low levels of a protein called…
Lifetime TV‘s October 2 special edition of “Behind the Mystery: Rare and Genetic” focuses on Friedreich’s ataxia (FA) through the stories of a woman living with the rare disease and a medical researcher studying it. “Behind the Mystery” educates viewers on rare and genetic disorders and seeks…
An initiative called the Friedreich’s Ataxia Accelerator has been launched by the Broad Institute of Massachusetts Institute of Technology (MIT) and Harvard University to develop therapeutic strategies for people with the disorder. Notably, the Accelerator will cultivate a community of researchers focused on understanding the molecular mechanisms…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
Team Gleason and CureDuchenne have teamed with Google’s Project Euphonia to make automated speech recognition more accessible to people with neurological disorders, including Friedreich’s ataxia. Although automated speech recognition increasingly powers voice-activated technologies like Google Assistant, it struggles to adapt to changes in speech that accompany certain…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), has issued a positive opinion about granting orphan drug status to CTI-1601, Larimar Therapeutics’ investigational treatment for Friedreich’s ataxia (FA). The company now is expecting…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…
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