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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Research News
An update on genetic testing in ataxia
There are many different types of ataxia, with numerous causes. For example, ataxia can be genetic, occur due to an autoimmune response (e.g. gluten ataxia) or when we do not
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Drug Repurposing to Accelerate Therapeutic Development for ITPR1-Associated Cerebellar Ataxias
Principal Researchers: Dr Ricardo Parolin Schnekenberg, Prof Joanne Ng, Prof Selina Wray and Prof Francesco Muntoni, University College London (UK) Scientific summary: Cerebellar ataxias are genetically heterogeneous and clinically
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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The Sheffield Ataxia Centre experience on the use of 3,4-diaminopyridine in SCA27BÂ
The team at the Sheffield Ataxia Centre has recently published a paper in the Journal of Neurology. To read the paper please click here. The paper details the Sheffield Ataxia Centre experience and gives some support to the potential use of 3,4-diaminopyridine as a therapy to alleviate symptoms
The Sheffield Ataxia Centre experience on the use of 3,4-diaminopyridine in SCA27B Read More »
IntraBio and levacetylleucine (AQNEURSA)Â updates
IntraBio is a biopharmaceutical company focused on developing treatments for progressive neurological conditions. This page provides updates on their levacetylleucine programme.  Levacetylleucine is a chemically modified version of the amino
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Using the SPAX mobile app for measuring symptom progression in the home in spastic ataxias
The European PROSPAX Consortium have shared results from their 8-week remote study of the SPAX smartphone app for tracking symptoms in spastic ataxias. About the PROSPAX project The PROSPAX consortium
VICO Therapeutics Clinical Trial updates
Spinocerebellar ataxias (SCAs) are a group of rare, progressive hereditary genetic disorders that affects the cerebellum, brain stem and spinal cord. Currently, more than 30 types of SCAs have been identified. SCAs are caused by genetic mutations, which lead to faulty
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Changes to the NICE cost-effectiveness thresholdÂ
In December 2025 the government announced that from April 2026 it will increase the standard NICE cost-effectiveness thresholds, which are used when evaluating new medicines for use in the NHS, from £20,000 – £30,000 to £25,000 – £35,000. Notably, the threshold has
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New paper published on the SARAhome scale to remotely assess ataxia symptoms in SCA3
In 2021, Ataxia UK co-funded a research project with the National Ataxia Foundation, ‘Assessment of ataxia severity under real-life conditions with SARAhome: A multicenter study in spinocerebellar ataxia type 3 (SCA3)’. This project was led by Dr
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Towards a personalised treatment for SCA1
In 2022, Ataxia UK awarded funding to Dr Francesca Salvatori at the University of Ferrera in Italy, for a project focused on SCA1. Previous work SCA1 is caused by the production of
Towards a personalised treatment for SCA1 Read More »
New paper published looking at peripheral and autonomic nervous system involvement in SCA3
Spinocerebellar ataxia type-3 (SCA3) is one of the most common types of SCA worldwide, and is characterised by progressive decline in daily function and quality of life. Whilst cerebellar ataxia
Ataxia UK and CureDRPLA provided feedback regarding the scope of a framework under development for individualised treatments in the UK
NHS England opened a consultation to seek feedback on an operational framework they are developing to support the future use of individualised genetic therapies in the NHS in England. They
MHRA publishes plans to reform medicines regulatory systems for rare diseases
Existing medicines regulatory processes do not consider the challenges that accompany treatment development for rare diseases. The regulatory body for medicines in the UK, the MHRA, has recognised this and is planning a major change by proposing a new framework for rare diseases. We welcome their report,
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Sharing results from Ataxia UK-funded project looking at potential treatment for SCA3
In a project funded by Ataxia UK, Dr Andreia Teixeira-Castro (pictured left) and her team at the University of Minho, Portugal, have investigated the therapeutic potential of the anti-depressant vortioxetine
Sharing results from Ataxia UK-funded project looking at potential treatment for SCA3 Read More »
Ataxia UK-funded project shows potential for SMaRT gene therapy to treat SCA1
This project, led by Prof Karen Anthony at the University of Northampton and Dr Ronald Buijsen at Leiden University Medical Centre, aimed to evaluate the potential of targeting RNA to
Ataxia UK-funded project shows potential for SMaRT gene therapy to treat SCA1 Read More »
Promising results from Ataxia UK co-funded project targeting SCA3 using nanobodies
Dr Sandra Macedo-Ribiero of the University of Porto, Portugal, has shared results from her project co-funded by Ataxia UK, ‘NanoSCA3: Development of brain-targeted nanobodies for application in spinocerebellar ataxia type
Promising results from Ataxia UK co-funded project targeting SCA3 using nanobodies Read More »
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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Watch the 2025 DRPLA Research Update on YouTube
We run the DRPLA Research Programme in partnership with CureDRPLA and every year we provide a research update for non-scientists. Watch the 2025 recap in which Dr Silvia Prades provides
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The Sheffield Ataxia Centre’s 2025 ataxia study day for healthcare professionals
Last month, on 7th November, the Ataxia UK-accredited Sheffield Ataxia Centre hosted an ataxia study day for healthcare professionals at the University of Sheffield, chaired by Consultant Neurologists Professor Marios Hadjivassiliou and Dr Priya Shanmugarajah, and organised
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Sharing results from Ataxia UK-funded project on restoring NKX6-2 gene function in Spastic Ataxia 8 cells in the lab
Dr Federico Herrera of the University of Lisbon, Portugal, has recently shared results of his Ataxia UK-funded project, ‘Restoring NKX6-2 function by protein complementation: a proof-of-concept’. Spastic ataxia 8 (SPAX8)
New paper published on TG6 antibodies and patient outcomes in Gluten-related neurological disorders
Gluten-related disorders, also known as gluten sensitivity disorders, happen when a person’s immune system reacts to gluten, and immune cells called autoantibodies, mistakenly attack the body’s tissues. This is called an autoimmune response. There are numerous autoantibodies that can be
New paper published on measuring treatment effectiveness in people with SCA3
Researchers Prof Bart van de Warrenburg, Dr Roderick Maas and Kristofoor Leeuwenberg at Radboud University Medical Center in the Netherlands have recently published a paper in the journal Cerebellum on
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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
Sharing results from Ataxia UK-funded project to aiming to model ARSACS in the lab
Dr Federico Herrera, based at the University of Lisbon, Portugal, has recently shared the results of his Ataxia UK-funded project, ‘Towards a pharmacological model of Autosomal Recessive Spastic Ataxia of
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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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