Design Therapeutics is Biotechnology company developing DT-216, which is a potential treatment for Friedreich’s ataxia (FA). FA is caused by a change to the frataxin gene. Genes are made […]
Design Therapeutics announce results from Phase 1 FA trial Read More »
Earlier this year, Ataxia UK awarded a travel grant to Dr David Pellerin at University College London, to attend the 2023 Annual Meeting of the American Academy of Neurology. Supporting
American Academy of Neurology 2023 Read More »
Biohaven is a pharmaceutical company developing Troriluzole as a treatment for SCA3. In May 2023, Biohaven submitted a New Drug Application (NDA) to the US regulators (the FDA), to apply
Biohaven update on Troriluzole programme Read More »
Do you want to learn about what gene therapy is, or what the future of gene therapy research may bring? Euro-ataxia, with support from Ataxia UK, will be hosting an
Gene therapy educational webinar Read More »
Larimar Therapeutics is a biotechnology company developing treatments for rare conditions, including Friedreich’s ataxia (FA). They are testing a treatment for FA called CTI-1601, which is a protein replacement therapy
Larimar Therapeutics receives FDA clearance to proceed with next stage of trial Read More »
Lexeo Therapeutics is a company developing gene therapy for people with FA cardiomyopathy. They recently announced the completion of the first cohort in their Phase I/II trial, testing their gene
Lexeo Therapeutics FA trial update Read More »
Biohaven is a pharmaceutical company with an ataxia research programme. They carried out a clinical trial testing Troriluzole in a number of SCAs. Results of this 48 week trial showed
Biohaven submits NDA to FDA Read More »
SCA-Remote is a study which aims to understand how different spinocerebellar ataxia (SCA) symptoms develop over time. You can read more about the study here. The researchers involved in this
SCA-Remote study recruiting people without ataxia Read More »
SIMPATHIC Consortium receives grant from European Commission The SIMPATHIC Consortium, led by the Dutch Radboud University Medical Center and Amsterdam University Medical Centers, has developed a new approach to expedite
8.8 million euros for accelerated drug repurposing for rare neurological disorders Read More »
Larimar Therapeutics is a biotechnology company, developing a treatment for Friedreich’s ataxia (FA) called CTI-1601. CTI-1601 replaces frataxin, which is the protein that is reduced in FA. Larimar Therapeutics recently
Larimar Therapeutics – FA trial update Read More »
Vico Therapeutics is a company developing therapies for neurological conditions, including some of the SCAs. In April 2023, they announced that they have dosed the first patient in their Phase
Vico Therapeutics – First patient dosed in trial Read More »
The optimal clinical pathway sets out what good treatment, care and support looks like for people with neurological conditions, from those experiencing the first symptoms, to those that have lived
Optimal clinical pathway for people in England with rare neurological conditions Read More »
Professor Anja Lowit (University of Strathclyde) and team have published positive results from their study of a new approach to speech therapy, called ClearSpeechTogether. In this Ataxia UK-funded project, people
Positive results from Ataxia UK-funded speech therapy project Read More »
Researchers in Canada have been studying a rehabilitation programme for a type of ataxia called ARSACS. The aim of their study was to see whether a rehabilitation programme could improve
Exploratory study – ARSACS exercise programme Read More »
Exciting research from two ground-breaking studies shows the power of wearable technology and artificial intelligence in monitoring movement disorders, including Friedreich’s ataxia (FA) Researchers have used a wearable tool to
Wearable technology for Friedreich’s ataxia Read More »
The 100,000 Genomes Project, launched in 2013, aimed to sequence 100,000 genomes from people with rare conditions, such as ataxia, or cancer. Sequencing the whole genome of people with rare
Did you take part in the 100,000 Genomes Project? Read More »
Design Therapeutics have announced positive data from the first part of their Phase I clinical trial of DT-216. They found that treatment with DT-216 increased frataxin mRNA. Frataxin mRNA is
Design Therapeutics announce positive data from Friedreich’s ataxia trial Read More »
ARSACS is a rare inherited ataxia caused by mutations in a gene called the SACS gene, which is responsible for producing the sacsin protein. In people with ARSACS, the sacsin
Understanding the cause of ARSACS Read More »
Rare neurological conditions, such as ataxia, often cause problems with balance and coordination, which can limit physical activity. Interventions aimed at improving levels of physical activity are often recommended, for
Measuring physical activity in trials Read More »
The Association of Medical Research Charities (AMRC), of which Ataxia UK is a member, created an animation describing the fundamental role that charities play in research in the UK. Charities
Making research happen Read More »
The EFACTS registry (European Friedreich’s Ataxia Consortium for Translational Studies) collects clinical information from people with Friedreich’s ataxia (FA) across Europe, including at the London Ataxia Centre. Ataxia UK provided
Agreement creates the largest worldwide database for Friedreich’s ataxia Read More »
Last year, we publicised two surveys from the British Paediatric Neurology Association (BPNA) and the James Lind Alliance (JLA). The aim of these surveys was to identify research priorities for
Top Ten UK research priorities for interventions in childhood neurological conditions Read More »
Between November 1st – 4th 2022, Ataxia UK, the National Ataxia Foundation (NAF), and the Friedreich’s Ataxia Research Alliance (FARA) co-hosted the International Congress for Ataxia Research (ICAR). This conference
International Congress for Ataxia Research 2022 Read More »
Everyone aged 5 (on or before 31 August 2022) and over can get a 1st and 2nd dose of the COVID-19 vaccine. There are also two booster doses of the
Covid-19 vaccine: Autumn 2022 Booster Read More »
A study led by researchers at the London Ataxia Centre, in collaboration with UC Davis California and Brunel University London, has shown that a combination of resveratrol and a multiple
Drug combination shows positive effect in models of FA Read More »
Researchers from The German Centre for Neurodegenerative Diseases (DZNE) in Tübingen, and the ESMI Consortium, have published a study looking at the potential for gait measurements to quantify progression of
Gait measurements as a potential biomarker for SCA3 Read More »
PROSPAX (PROgression chart of SPAstic ataXias) and TREAT-ARCA are two global projects, aimed at advancing research into specific ataxias (ARSACS and SPG7 for PROSPAX, and ARSACS and COQ8A-ataxia for TREAT-ARCA).
PROSPAX and TREAT-ARCA project updates Read More »
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 2022 Read More »
Dr Hector Garcia-Moreno is a Neurologist and Clinical Research Fellow working at the London Ataxia Centre, in the team led by Professor Paola Giunti. Dr Garcia-Moreno is involved in the
Identification of SCA3 biomarkers – an interview with Dr Hector Garcia-Moreno Read More »
LEXEO Therapeutics is a gene therapy company, developing treatments for a number of conditions including cardiomyopathy associated with FA. In February 2022, LEXEO Therapeutics announced that the FDA had cleared
LEXEO Therapeutics announce plans for clinical trial Read More »
