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 […]
Lexeo Therapeutics update page Read More »
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
Developing audio messages to help those with ataxia when making calls Read More »
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 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 »
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
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 »
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
Changes to the NICE cost-effectiveness threshold Read More »
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 »
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
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
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,
MHRA publishes plans to reform medicines regulatory systems for rare diseases Read More »
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 »
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 »
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 »
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.
A new project studying neurostimulation in ataxia Read More »
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
Watch the 2025 DRPLA Research Update on YouTube Read More »
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
The Sheffield Ataxia Centre’s 2025 ataxia study day for healthcare professionals Read More »
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)
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
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
New paper published on measuring treatment effectiveness in people with SCA3 Read More »
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
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 »
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 »
 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 »
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 »
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 »
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.
Reflections on the 2025 Euro-ataxia conference 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 »
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
Managing sleep and fatigue symptoms in ataxias Read More »
