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 […]
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
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,
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
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 »
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 »
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 »
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.
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 »
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 »
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 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
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 »
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 »
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
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.
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 »
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 »
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
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 »
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
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
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
In this study, researchers at the University of Southampton looked at the impact of ataxia symptoms on self-reported mental wellbeing in adults in the UK. They compared findings, collected from
Paper published on the impacts of ataxia symptoms on mental health Read More »
Euro-ataxia, of which Ataxia UK is an active member, is the federation of 22 European ataxia patient organisations. The 2024 Euro-ataxia conference took place in London on 15th-16th November, organised
Ataxia patient organisations united at the 2024 Euro-ataxia annual conference Read More »
The International Congress for Ataxia Research (ICAR) 2024 took place in London from 12th – 15th November. It was organised by Ataxia UK, the National Ataxia Foundation, the Friedreich’s Ataxia
Highlights of the International Congress for Ataxia Research 2024 Read More »
