Key takeaways from the webinar include the complexity of the relationship between FA patients and caregivers, and the importance of practicing open communication and empathy and asking for help when necessary.
PTC Therapeutics says it’s planning, by this December, to ask the U.S. Food and Drug Administration (FDA) to approve its oral therapy vatiquinone — which has been tested in pediatric patients as well as adults — as a treatment for Friedreich’s ataxia (FA). “We look forward to…
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…
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,…
A low dose of calcitriol, an activated form of vitamin D used to help manage hypoparathyroidism and other disorders, led to statistically significant increases in frataxin protein levels among people with Friedreich’s ataxia in a small clinical trial conducted in Spain. Although patients in the yearlong study did…
Thanks to a philanthropic donation of up to £650,000 (about $825,000), the Oxford-Harrington Rare Disease Centre (OHC) is offering two new grant programs to support the development of treatments for Friedreich’s ataxia (FA). The new awards, dubbed The FA Alliance Catalyst Fund and The FA Alliance Innovation Fund,…
An experimental compound called ATH434, currently being evaluated in clinical trials for a neurological disorder called multiple system atrophy, also may have the potential to treat Friedreich’s ataxia (FA). Data specifically indicate that ATH434 could help to reduce the toxic buildup of iron in cells that is characteristic…
A deficiency of frataxin, the protein whose lack causes Friedreich’s ataxia, leads to problems with mitochondrial translation — the molecular process by which new proteins are made in the mitochondria, the so-called powerhouse of the cell responsible for energy production — according to a new study. The findings specifically…
The European Commission has approved Skyclarys (omaveloxolone) for people with Friedreich’s ataxia (FA) ages 16 and older, making the treatment the first to win approval for FA in the European Union. The decision comes just a few months after a committee of the European Medicines Agency (EMA) issued…
People in the Friedreich’s ataxia (FA) community await the possibility of gene therapies for their disease, with many saying they would participate in gene therapy trials regardless of the potential for side effects or other consequences. Around half of the survey’s 137 respondents expected that such treatment — delivering…
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