News

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…

When present at levels 20 times higher than normal, frataxin became toxic and caused heart problems in a mouse model of Friedreich’s ataxia (FA), a study found. According to the study authors, these findings underscore the importance of selecting an appropriate viral vector for gene therapy to ensure…

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…

The natural antioxidant epicatechin significantly improves heart structure and function in young people with Friedreich’s ataxia (FA) over nearly six months of treatment, a small Phase 2 study has shown.  The findings support the need for larger trials that include…

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…

Simultaneous suppression of two histone methyltransferases (HMTs) significantly increased the activity of the FXN gene in cells derived from people with Friedreich’s ataxia (FA) and in a mouse model of the disease. However, this boost in FXN activity failed to raise the levels of its resulting protein, frataxin,…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

Reata Pharmaceuticals, along with a group of key opinion leaders and the Friedreich’s Ataxia Research Alliance (FARA), have proposed the launch of a study to confirm the effectiveness of omaveloxolone (RTA 408), the company’s investigational oral therapy for Friedreich’s ataxia (FA). If accepted by the…