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.Â
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 »
On 8th October 2024, the pharmaceutical company PTC Therapeutics announced positive results from their phase III trial of their drug Vatiquinone for adults and children with Friedreich’s ataxia (FA). Analysis
Recently, our research intern Scarlett Parr-Reid sat down with Lucie Hogger, Speech and Language Therapist at the London Ataxia Centre to talk about the speech and language therapy services offered
On 25th September 2024, the pharmaceutical company Intrabio announced that the FDA in the US has approved their small molecule drug Levacetylleucine for the treatment of Niemann-Pick Disease Type C
On September 23rd, 2024, the pharmaceutical company Biohaven announced positive topline results from their study of the drug Troriluzole to treat SCAs 1,2,3,6,7,8 and 10. Â Â Those with SCA who
On September 13th, the pharmaceutical company Vico Therapeutics announced positive interim phase 1/2a clinical data of VO659 in the treatment of Huntington’s disease. VO659 demonstrated a 28% average reduction in
Ataxia UK has worked closely with the London Ataxia Centre researchers and other experts on a research study assessing the impact of specialist ataxia centres in Europe. This was part
Our Research Intern, Scarlett Parr-Reid, sits down with Dr. Federico Herrera (pictured below) for the second part of a two-part interview series, to discuss his team’s research project at the
In conversation with Dr. Federico Herrera part 2 Read More »
Many people with ataxia do not have a specific genetic diagnosis and the cause of their ataxia is unknown. In 2023, two papers were published which identified a genetic mutation
Update on genetic testing for SCA27B Read More »
On 23rd July, Ataxia UK hosted a webinar and Q&A on the UK approval process for Omaveloxolone (Skyclarys) in adults with FA. This was in partnership with the consultancy Realise
Ataxia UK hosts Webinar on UK approval process for Skyclarys in adults with FA Read More »
Lexeo Therapeutics is a pharmaceutical company developing a gene therapy called LX2006 for people with Friedreich’s ataxia (FA) hypertrophic cardiomyopathy (disease of the heart involving stretching, thickening, or stiffening of
On 12th July Ataxia UK hosted a webinar on how medicines get approved for use in the UK and how decisions are made on their funding in the NHS. The
Ataxia UK hosts an educational webinar on the UK medicines approval process Read More »
Our research intern, Scarlett Parr-Reid, sits down with Dr Ng to discuss his research into mitochondrial disease and spinocerebellar ataxia type-6 (SCA6). Tell me a bit about yourself and your
Our research intern, Scarlett Parr-Reid, sits down with Dr. Herrera to discuss his research on spastic ataxia type-8 (SPAX8). Can you share a little about yourself, your background as a
The PROSPAX Consortium has recently published two new research papers as part of the PROSPAX project, an international project that aims to better understand how spastic ataxias progress over time
PROSPAX Consortium publishes two new research papers Read More »
Rachel Higgins (BSc (Hons) MCSP) is a Clinical Specialist Neurological and Vestibular Physiotherapist at The National Hospital for Neurology and Neurosurgery (NHNN). She has worked within the field of neurology
In conversation with London Ataxia Centre Specialist Physiotherapist, Rachel Higgins Read More »
Ataxia UK started a partnership with CureDRPLA in March 2020 with the mission of advancing research for the very rare ataxia Dentatorubral-Pallidoluysian Atrophy (DRPLA) and finding potential treatments. With funding
