In adults with Friedreich’s ataxia (FA), treatment with nomlabofusp may increase levels of frataxin protein to at least…
Articles by Marisa Wexler, MS
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…
People with Friedreich’s ataxia often show abnormalities in their eye movements and looking for characteristic changes there may help…
Design Therapeutics is on track to start clinical trials next year of its new and improved experimental treatment for…
A low dose of calcitriol, an activated form of vitamin D used to help manage hypoparathyroidism and other disorders,…
Thanks to a philanthropic donation of up to £650,000 (about $825,000), the Oxford-Harrington Rare Disease Centre (OHC) is offering…
An experimental compound called ATH434, currently being evaluated in clinical trials for a neurological disorder called multiple system atrophy,…
A deficiency of frataxin, the protein whose lack causes Friedreich’s ataxia, leads to problems with mitochondrial translation — the…
The European Commission has approved Skyclarys (omaveloxolone) for people with Friedreich’s ataxia (FA) ages 16 and older, making…