In people with FA, a protein called frataxin is reduced. SGT-212, developed by pharmaceutical company Solid Biosciences, is designed to deliver frataxin to both the cerebellum in the brain, and […]
Solid Biosciences and SGT-212 gene therapy updates Read More »
Dr Rosella Abeti and Prof Paola Giunti at the London Ataxia Centre, UCL Institute of Neurology, have shared results from their project which was funded by Ataxia UK. The project was titled ‘Exploring novel iron-mediated mechanisms to prevent cellular death in
Heart problems can be a symptom of Friedreich’s ataxia (FA). In 2022, Prof Richard Festenstein of Imperial College London received a grant from Ataxia UK to generate a heart cell
Researchers identify potential new biomarker of FA disease progression Read More »
The pharmaceutical company Lexeo Therapeutics has announced positive updates from their phase I/II trial of the gene therapy LX2006 to treat cardiomyopathy in Friedreich’s ataxia (FA).  Interim data has shown
Lexeo shares positive data in trial of LX2006 for FA cardiomyopathy Read More »
Ataxia UK has funded a number of research projects in Friedreich’s ataxia (FA) by Dr Sara Anjomani-Virmouni, a researcher based at Brunel University, London. As a result of this funded
Antioxidants as a potential therapy for Friedreich’s ataxia Read More »
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
