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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Cerebellar ataxia
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
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
IntraBio and levacetylleucine (AQNEURSA) updates Read More »
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
VICO Therapeutics Clinical Trial updates Read More »
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
New paper published on the SARAhome scale to remotely assess ataxia symptoms in SCA3 Read More »
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
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 »
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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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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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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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
Sharing results from Ataxia UK-funded project to aiming to model ARSACS in the lab Read More »
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
Reflections on the 2025 Clinical Trials in Ataxias Conference Read More »
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
Pre-implantation genetic testing approved to diagnose SCAN3 Read More »
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
FDA does not approve new drug application for troriluzole for spinocerebellar ataxias Read More »
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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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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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
Intrabio to conduct phase III trial in SCA6 and Episodic Ataxia Type-2 Read More »
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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Arrowhead Pharmaceuticals starts recruitment for SCA2 trial
The pharmaceutical company Arrowhead Pharmaceuticals has begun recruitment for their phase 1 randomised placebo-controlled trial of ARO-ATXN2 in adults with SCA2. The programme is a partnership with the pharmaceutical company
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New paper published on promising speech therapy programme for people with Cerebellar Multiple System Atrophy
Prof Anja Lowit, Speech and Language Therapist based Strathclyde University, Glasgow, together with Prof Marios Hadjivassiliou from the Sheffield Ataxia Centre have published a new research paper looking at a
Transcranial brain stimulation can improve symptoms in SCA3
A recent paper published in the scientific journal Cell Reports Medicine has shown that a type of non-invasive brain stimulation called transcranial alternating current stimulation (tACS) provides benefits to people
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Reflections from the International Drug Repurposing Conference 2025
On 7th and 8th May, Ataxia UK’s Research Communications Officer, Scarlett Parr-Reid, attended Beacon for Rare Diseases and Remedi4All’s second International Drug Repurposing Conference (IDR25), held in Amsterdam.  Drug repurposing
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Biohaven withdraws Troriluzole application to EMA for Spinocerebellar ataxias
In March, Biohaven shared that they have taken the decision to withdraw their new drug application to the European Medicines Agency (EMA) for Troriluzole to treat adults with all Spinocerebellar
Biohaven withdraws Troriluzole application to EMA for Spinocerebellar ataxias Read More »