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
Celebrating the impact of Ataxia UK-funded research Read More »
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
Arrowhead Pharmaceuticals starts recruitment for SCA2 trial Read More »
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
PTC Therapeutics announces that the FDA has not approved Vatiquinone to treat FA in adults and children
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
Co-designing audio support messages with and for people with ataxia: a telephone version of the “I’m not drunk” ID card
Upcoming project: Co-designing audio support messages with and for people with ataxia: a telephone version of the “I’m not drunk” ID card Principal Investigators: Dr Maria Cairney and Prof Anja
Subtyping Hereditary Cerebellar Ataxias based on multimodal disease progression patterns Subtyping Hereditary Cerebellar Ataxias based on multimodal disease progression patterns
Upcoming project: Subtyping Hereditary Cerebellar Ataxias based on multimodal disease progression patterns Subtyping Hereditary Cerebellar Ataxias based on multimodal disease progression patterns Principal investigators: Dr Susmita Saha, Prof Nellie Georgiou-Karistianis,
Investigating The Molecular Mechanisms of SCA2 Using 3D Cerebellar Organoids and Multi-Omics Approaches: A Platform for Advancing Ataxia Research
Upcoming project: Investigating The Molecular Mechanisms of SCA2 Using 3D Cerebellar Organoids and Multi-Omics Approaches: A Platform for Advancing Ataxia Research Principal applicants: Prof Jorgen Erik Nielsen, Senior Researcher Patrick
NfL may be a potential biomarker in Friedreich’s ataxia
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 »
Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia
Title: Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia Principal researchers: Boubker Zaaimi (Aston University, UK), Prof Stuart Baker (Newcastle University, UK), Prof Mark Baker (Newcastle University, UK)
Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia Read More »
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
Transcranial brain stimulation can improve symptoms in SCA3 Read More »
Antioxidants as a potential therapy for Friedreich’s ataxia
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 »
Caring for people with progressive ataxia – lived experience of family carers and co-development of support mechanisms
Caring for people with progressive ataxia – lived experience of family carers and co-development of support mechanisms Principal researchers: Prof Anja Lowit and Prof Christopher Graham, Strathclyde University, Scotland.
Ataxia UK and Realise Advocacy host webinar on access to omaveloxolone for adults aged 16 and over with FA in the UK
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
Sign our letter to the UK Secretary of State for Health and Social Care requesting interim access to omaveloxolone for FA patients in the UK
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)
Living with Late Onset – Exercise and FA
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 »
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
Reflections from the International Drug Repurposing Conference 2025 Read More »
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 »
Biogen announces plans to start their global phase 3 trial of Omaveloxolone in children aged 2-15, with sites in the UK
Biogen have shared their plan to initiate their BRAVE study, a global phase 3 trial of Omaveloxolone (SKYCLARYS®) in children aged 2-15 years. The trial will study the effects and
MHRA approves Omaveloxolone for Friedreich’s ataxia
The UK medicines regulator (MHRA) approves the first treatment for Friedreich’s ataxia (FA), Omaveloxolone (Skyclarys). On 23 April 2025, the MHRA approved Omaveloxolone for the treatment of those aged 16
MHRA approves Omaveloxolone for Friedreich’s ataxia Read More »
Living with Late Onset – Clare’s Friedreich’s ataxia Story
Two years ago, at the age of 44, I was diagnosed with a rare condition called Friedreich’s Ataxia (FA). I had balance and speech symptoms for a few years and
Living with Late Onset – Clare’s Friedreich’s ataxia Story Read More »
New study published on goal-directed Rehabilitation vs. Standard Care in those with hereditary Cerebellar ataxia
A study led by Dr Sarah Milne at the Murdoch Children’s Research Institute, Australia, has recently been published in the journal ANNALS of Neurology. The study looked at the impact
Lexeo Therapeutics provides updates on their gene therapy programme for Friedreich’s ataxia cardiomyopathy
On March 24th 2025, the pharmaceutical company Lexeo Therapeutics provided a number of updates on their phase 1/2 trial of the gene therapy drug LX2006 for Friedreich’s ataxia (FA) cardiomyopathy.
Larimar Therapeutics announce plans for phase 3 trial of Nomlabofusp for FA
On March 24th 2025, the pharmaceutical company Larimar Therapeutics announced their plans to start conducting a phase 3 trial of their Friedreich’s ataxia (FA) drug Nomlabofusp in mid-2025. This follows
Larimar Therapeutics announce plans for phase 3 trial of Nomlabofusp for FA Read More »
Solaxa announces plan for phase 3 clinical trial for SCA27B
In March, the pharmaceutical company Solaxa has announced plans to conduct a phase 3 clinical trial of their drug SLX-100 (repurposed 4-aminopyridine) for Spinocerebellar ataxia 27B (SCA27B). The trial will
Solaxa announces plan for phase 3 clinical trial for SCA27B Read More »
Ataxia UK hosts webinar on managing ataxia symptoms, presented by Dr. Michael Parkinson of the London Ataxia Centre
On Wednesday 5th February, Ataxia UK hosted a webinar and Q&A as part of Wellbeing Week, a week from 3rd – 7th February dedicated to providing support and information to
Cure Rare Disease receives funding for SCA3 gene therapy trial
On February 6th, the US not-for-profit biotechnology company Cure Rare Disease announced that they have been awarded 5.69 million dollars in funding from the Californian Institute of Regenerative Medicine (CIRM)
Cure Rare Disease receives funding for SCA3 gene therapy trial Read More »
Alterity Therapeutics announces positive results from its phase 2 trial of the drug ATH434 in MSA
On January 30th 2025, the pharmaceutical company Alterity Therapeutics announced positive data from their phase 2 placebo-controlled trial in early-stage MSA. MSA is a progressive neurological condition leading to impaired
In Conversation with Dr. Alanna Watt, lead of the Watt Lab at McGill University, Canada, on her research looking at changes in the brain in SCA6
Dr. Alanna Watt is an Associate Professor in the department of biology at McGill University, Canada, and has been running the Watt Lab since 2011. The Watt Lab focuses on
PROSPAX Consortium publishes results from their 4-year project on spastic ataxias, with more to come
About the PROSPAX project The PROSPAX consortium of patient groups, researchers and clinicians across Europe and Canada (including the UK), set up the PROSPAX project to study the progression of