Larimar Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) this year to grant accelerated approval to…
Articles by Marisa Wexler, MS
Errors during repair when DNA gets damaged may give rise to the mutations that cause Friedreich’s ataxia, a study…
The U.S. Food and Drug Administration (FDA) has given Solid Biosciences the go-ahead to start clinical testing of…
In adults with Friedreich’s ataxia (FA), treatment with nomlabofusp may increase levels of frataxin protein to at least…
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…