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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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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Towards improved mental quality of life in ataxias: Characterising psychological function and its correlates
Principal researcher:Â Dr Louisa Selvadurai, Monash University (Australia)Â Scientific Summary:Â Individuals with ataxia often experience cognitive and emotional challenges, such as low mood and word-finding difficulties (psychological symptoms), in addition to
Efficient and Personalised Speech Recognition for Ataxia
Principal Researcher: Prof Chenghua Lin, University of Manchester (UK) Scientific summary: The primary purpose of this investigation is to address the communication barriers experienced by individuals with ataxia by developing
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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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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
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
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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
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
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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
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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 »
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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Feasibility testing a double-blinded randomised cross-over trial as an independent evaluation of use of the Exopulse Mollii electrostimulation suit for people with ataxia
Principal researcher: Dr Lisa Bunn, Plymouth University (UK) Â Scientific Summary: This project directly responds to the needs expressed by people with ataxia for non-invasive treatments to improve daily life.
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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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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Pre-implantation genetic testing approved to diagnose SCAN3
The Human Fertilisation and Embryology Authority (HFEA), the UK’s independent regulator of fertility treatment and research using human embryos, has licensed Pre-implantation Genetic Testing (PGT) to diagnose a rare form of
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FDA does not approve new drug application for troriluzole for spinocerebellar ataxias
In September 2024 Biohaven announced positive results from their study of troriluzole to treat SCA 1, 2, 3, 6, 7, 8 and 10. Following these results they submitted a new
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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
Intrabio to conduct phase III trial in SCA6 and Episodic Ataxia Type-2
The pharmaceutical company Intrabio, who developed the recently FDA-approved drug AQNEURSA for people with Niemann-Pick Disease Type C, has announced their plans to begin a phase III trial of the
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