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
The pharmaceutical company Solid Biosciences announced in December that their gene therapy for Friedreich’s ataxia (FA), known as SGT-212, has been granted Rare Pediatric Disease and Fast Track designations by
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 »
Ataxia UK’s Internal Ethics Committee is recruiting new members. This role involves attending regular online Committee meetings to give feedback on material to be used for ataxia research. This includes
Ataxia UK’s research team is looking for contributors 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 »
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 »
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
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 »
Current clinical rating scales to measure disease progression in people with spasticity and ataxia are focused solely on either spasticity symptoms (stiffness of muscles leading to involuntary spasms) or ataxia
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
Celebrating the impact of Ataxia UK-funded research Read More »
The pharmaceutical company Arrowhead Therapeutics 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
Arrowhead Pharmaceuticals to open UK sites in SCA2 trial Read More »
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
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
Scientists created a mouse model with the genetic change that causes DRPLA in people. These mice showed symptoms common of DRPLA, such as balance problems, seizures, tremors, and poor coordination.
Genetic treatment protects against DRPLA symptoms in a mouse model with DRPLA Read More »
As part of our partnership with CureDRPLA we have been building connections with the scientific and clinical community in Japan because DRPLA, a rare form of ataxia, is more prevalent
Strengthening ties with Japan’s rare disease community Read More »
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
NfL may be a potential biomarker in Friedreich’s ataxia Read More »
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
Transcranial brain stimulation can improve symptoms in SCA3 Read More »
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
Antioxidants as a potential therapy for Friedreich’s ataxia Read More »
On 4th June, 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 DT-216P2.
First FA patient dosed in RESTORE-FA trial Read More »
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
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)
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
Living with Late Onset – Exercise and FA Read More »
