On 4th June, the Biotechnology company Design Therapeutics announced that they have dosed the first person with Friedreich’s ataxia (FA) in their phase 1 clinical trial of their drug DT-216P2. […]
First FA patient dosed in RESTORE-FA trial Read More »
Two years ago, at the age of 44, I was diagnosed with a rare condition called Friedreich’s Ataxia (FA). I had balance and speech symptoms for a few years and
Living with Late Onset – Clare’s Friedreich’s ataxia Story Read More »
A study led by Dr Sarah Milne at the Murdoch Children’s Research Institute, Australia, has recently been published in the journal ANNALS of Neurology. The study looked at the impact
On 26th February, the biotechnology company Voyager Therapeutics announced that as part of their collaboration with the biopharmaceutical company Neurocrine Biosciences, they have selected a lead development candidate in their
Findings from a recent study, ‘Health-related quality of life in Friedreich Ataxia: A study of impacts on patients and caregivers’, were presented at the International Society for Pharmacoeconomics and Outcomes
