Research Impact Report 2024
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 2024 Read More »
Friedreich’s ataxia
PTC Therapeutics announces positive updates from their phase III trial of Vatiquinone in FA
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
In conversation with the London Ataxia Centre Speech and Language Therapist, Lucie Hogger
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
Satisfaction is significantly higher in those attending UK specialist ataxia centres compared with those who do not, new paper shows
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
Ataxia UK hosts Webinar on UK approval process for Skyclarys in adults with FA
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
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Lexeo announces positive results from their phase 1/2 clinical trial programme for FA cardiomyopathy
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
Ataxia UK hosts an educational webinar on the UK medicines approval process
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
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In conversation with London Ataxia Centre Specialist Physiotherapist, Rachel Higgins
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 »
Larimar Therapeutics announces progress of the Nomlabofusp (CTI-1601) trial for Friedreich’s ataxia
Following clearance by the US drug regulatory body the Food and Drug Administration (FDA) in July 2023, the pharmaceutical company Larimar Therapeutics has completed full enrolment and dosing of the
In Conversation with London Ataxia Centre Consultant Neurologist, Professor Paola Giunti
Professor Paola Giunti is an award-winning Consultant Neurologist. She has specialised in ataxias for over 30 years, and set up the London Ataxia Centre in 2005. At the London Ataxia
In conversation with London Ataxia Centre Specialist Ataxia Nurse, Suzanne Booth
Suzanne has worked as a neurology specialist nurse for 19 years, supporting people with a range of long-term neurological conditions. She has recently completed an MSc in Advanced Clinical Practice,
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Alterity Therapeutics presents data on the potential of ATH434 to treat FA
The biotechnology company Alterity Therapeutics presented data from their lead Friedreich’s ataxia (FA) drug candidate ATH434 at the 2024 US World Orphan Drug Congress. In FA, a genetic change in
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SBT-589 may be cardioprotective in FA
On March 19th, the pharmaceutical company Stealth BioTherapeutics presented data on their molecule called SBT-589 in preclinical models of Friedreich’s ataxia (FA) at the Wellcome Trust Conference on Mitochondrial Medicine-Therapeutic
SBT-589 may be cardioprotective in FA Read More »
Voyager Therapeutics and Neurocrine Biosciences announce selection of a lead gene therapy candidate for their FA program
On 26th February, the biotechnology company Voyager Therapeutics announced that as part of their collaboration with the biopharmaceutical company Neurocrine Biosciences, they have selected a lead development candidate in their
Quality of care is higher in Specialist Ataxia Centres, European study finds
Ataxia UK 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 of
Quality of care is higher in Specialist Ataxia Centres, European study finds Read More »
New study shows impacts of FA on daily life, wellbeing, and relationships in those with FA and their caregivers Â
Findings from a recent study, ‘Health-related quality of life in Friedreich Ataxia: A study of impacts on patients and caregivers’, were presented at the International Society for Pharmacoeconomics and Outcomes
Positive news regarding Skyclarys® approval in the EU
Ataxia UK is delighted with today’s announcement from the EMA’s Committee for Medicinal Products for Human Use (CHMP), the regulatory body that makes recommendations on drug approvals in the EU.
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Paper published on study of effectiveness of omaveloxolone for Friedreich’s ataxia using natural history study data
The natural history of Friedreich’s ataxia (FA) has been studied as part of a project called the Friedreich’s ataxia Clinical Outcome Measures Study (FACOMS). Natural history studies track the course
Results of Ataxia UK-funded study assessing speech therapy in people with progressive ataxias
Research by Prof Anja Lowit and her team at Strathclyde University, Glasgow, studying the effectiveness of a new type of speech therapy for some progressive ataxias, has shown some promising
Update from PTC Therapeutics on their MOVE-FA programme for Friedreich’s ataxia
Following results from their MOVE-FA phase 3 clinical trial of the drug vatiquinone in children and adults with Friedreich’s Ataxia (FA), the pharmaceutical company PTC Therapeutics wrote to the US
Update from PTC Therapeutics on their MOVE-FA programme for Friedreich’s ataxia Read More »
Reflections on the Ataxia UK Annual Conference 2023
On Friday 20th and Saturday 21st October 2023, Ataxia UK held their first ever hybrid in-person and virtual annual conference for families affected by ataxia. The conference, which took place
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University student joins Friedreich’s ataxia research lab following his sister’s FA diagnosis
In this article, Owen Gittins, a 3rd year undergraduate student in biochemistry at the University of Cambridge, shares his journey into and involvement in Friedreich’s ataxia (FA) research. My
Euro-ataxia hosts first in a series of webinars with Ataxia UK on gene therapy  
On 19th September, Euro-ataxia hosted its first webinar on gene therapy as part of a new series with Ataxia UK. 70 audience members attended live, and 70 watched the recording.
Euro-ataxia hosts first in a series of webinars with Ataxia UK on gene therapy   Read More »
Design Therapeutics announce results from Phase 1 FA trial
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
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Gene therapy educational webinar
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 receives FDA clearance to proceed with next stage of trial
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
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Lexeo Therapeutics FA trial update
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 »
Results from PTC Therapeutics Friedreich’s ataxia trial
PTC Therapeutics have announced the initial results of their Phase 3 MOVE-FA trial of vatiquinone (PTC-743) in people with FA. Vatiquinone targets an enzyme called 15-lipoxygenase, which is a regulator
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Preliminary results of Larimar Therapeutics FA trial show increase in frataxin levels
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. More specifically, CTI-1601
Preliminary results of Larimar Therapeutics FA trial show increase in frataxin levels Read More »
Larimar Therapeutics – FA trial update
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
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