Research Impact Report 2026
The research that Ataxia UK funds has a real impact on the world of ataxia research, and our understanding of all ataxias. Every year we ask Ataxia UK-funded researchers to […]
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Friedreich’s ataxia
Design Therapeutics RESTORE-FA trial updates page
On 4th June 2025, 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
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Vitamin D levels and Friedreich’s ataxia
This research was carried out by Prof Giunti and her team at the London Ataxia Centre at the National Hospital for Neurology and Neurosurgery using the London arm of the
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Lexeo Therapeutics update page
Lexeo Therapeutics is a genetic medicine company, developing treatments for a number of conditions including cardiomyopathy associated with Friedreich’s ataxia (FA). Cardiomyopathy is a heart condition involving stretching, thickening, or stiffening of the heart muscle, making it harder to
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Developing audio messages to help those with ataxia when making calls
In an Ataxia UK funded project, Dr Maria Cairney and Prof Anja Lowit at the University of Strathclyde are developing audio messages to support people with ataxia whilst making phone
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A new project studying neurostimulation in ataxia
This study, based at the Institute of Neurology, University College London, is led by Neuroscientist Dr Tim West, and funded by the Royal Academy of Engineering and the Rosetrees Trust.
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Solid Biosciences and SGT-212 gene therapy updates
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
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Ataxia UK-funded project shows that mitochondrial proteins may be a potential target to treat cellular stress in Friedreich’s ataxia
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
Researchers identify potential new biomarker of FA disease progression
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
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Reflections on the 2025 Clinical Trials in Ataxias Conference
 On October 27th-28th 2025, representatives from Ataxia UK attended the first Clinical Trials in Ataxia (CTAX) conference in Amsterdam. CTAX was organised by Ataxia UK, the National Ataxia Foundation
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Reflections on the 2025 Euro-ataxia conference
On October 28th-29th 2025, Euro-ataxia hosted the International Ataxia Patient Conference in Amsterdam. This two-day event was attended by representatives from 18 ataxia patient organisations from Europe and North America.
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Lexeo shares positive data in trial of LX2006 for FA cardiomyopathy
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
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Managing sleep and fatigue symptoms in ataxias
As part of the Ataxia UK 2025 virtual annual conference, Emma Foster, Clinical Nurse Specialist at the Sheffield Ataxia Centre (pictured to the left) gave a talk on managing sleep
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Larimar Therapeutics Announces positive results from their phase II trial of Nomlabofusp in FA
On 29th September 2025, the pharmaceutical company Larimar Therapeutics announced positive results from their phase II trial of the drug Nomlabofusp for FA. Nomlabofusp is designed to increase levels of
Research Impact Report 2025
The research that Ataxia UK funds has a real impact on the world of ataxia research, and our understanding of all ataxias. Every year we ask Ataxia UK-funded researchers to
Research Impact Report 2025 Read More »
Celebrating the impact of Ataxia UK-funded research
Ataxia UK funds research that aims to find treatments and cures for the ataxias. This would not be possible without your continued generosity in donating to support this mission. Thank
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PTC Therapeutics announces that the FDA has not approved Vatiquinone to treat FA in adults and children
On 19th August 2025, the pharmaceutical company PTC Therapeutics announced the disappointing news that the US drug regulators, the FDA, have not approved the drug vatiquinone to treat adults and
NfL may be a potential biomarker in Friedreich’s ataxia
New research that stemmed from a collaboration between the London Ataxia Centre and the Children’s Hospital of Philadelphia has identified the raise of a protein called NfL, as a potential
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Antioxidants as a potential therapy for Friedreich’s ataxia
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
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Ataxia UK and Realise Advocacy host webinar on access to omaveloxolone for adults aged 16 and over with FA in the UK
On Friday 23rd May, Ataxia UK hosted a webinar and Q&A on access to omaveloxolone for adults with Friedreich’s ataxia (FA) in the UK. This webinar was presented in partnership
Sign our letter to the UK Secretary of State for Health and Social Care requesting interim access to omaveloxolone for FA patients in the UK
We recently shared with the community the disappointing news about that Biogen have withdrawn their application to NICE (the body that makes decisions on reimbursement of medicines in the UK)
Living with Late Onset – Exercise and FA
As FA progresses, I am getting more and more fearful about trying to go out for short walks. I know I am putting on weight. I bought a static bike/cross-trainer
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Biogen announces plans to start their global phase 3 trial of Omaveloxolone in children aged 2-15, with sites in the UK
Biogen have shared their plan to initiate their BRAVE study, a global phase 3 trial of Omaveloxolone (SKYCLARYS®) in children aged 2-15 years. The trial will study the effects and
MHRA approves Omaveloxolone for Friedreich’s ataxia
The UK medicines regulator (MHRA) approves the first treatment for Friedreich’s ataxia (FA), Omaveloxolone (Skyclarys). On 23 April 2025, the MHRA approved Omaveloxolone for the treatment of those aged 16
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Living with Late Onset – Clare’s Friedreich’s ataxia Story
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
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New study published on goal-directed Rehabilitation vs. Standard Care in those with hereditary Cerebellar ataxia
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
Lexeo Therapeutics provides updates on their gene therapy programme for Friedreich’s ataxia cardiomyopathy
On March 24th 2025, the pharmaceutical company Lexeo Therapeutics provided a number of updates on their phase 1/2 trial of the gene therapy drug LX2006 for Friedreich’s ataxia (FA) cardiomyopathy.Â
Larimar Therapeutics announce plans for phase 3 trial of Nomlabofusp for FA
On March 24th 2025, the pharmaceutical company Larimar Therapeutics announced their plans to start conducting a phase 3 trial of their Friedreich’s ataxia (FA) drug Nomlabofusp in mid-2025. This follows
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Ataxia UK hosts webinar on managing ataxia symptoms, presented by Dr. Michael Parkinson of the London Ataxia Centre
On Wednesday 5th February, Ataxia UK hosted a webinar and Q&A as part of Wellbeing Week, a week from 3rd – 7th February dedicated to providing support and information to
Paper published on the impacts of ataxia symptoms on mental health
In this study, researchers at the University of Southampton looked at the impact of ataxia symptoms on self-reported mental wellbeing in adults in the UK. They compared findings, collected from
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