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 »
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
On October 26th 2023, the pharmaceutical company Biohaven submitted an application for a new drug approval of Troriluzole to the European Medicines Agency (EMA) for the treatment of Spinocerebellar Ataxia
Biohaven submits application to the EMA for a new drug approval of Troriluzole for SCA3 Read More »
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.
Positive news regarding Skyclarys® approval in the EU Read More »
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
On 1st December 2023, the pharmaceutical company Seelos Therapeutics announced they had stopped their phase 2/3 programme known as STRIDES, which had been investigating a new drug for Spinocerebellar Ataxia
Seelos Therapeutics has stopped their phase 2/3 trial of Trehalose for SCA3 Read More »
The 7th International Symposium on Autosomal Spastic Recessive Ataxia of Charlevoix-Saguenay (ARSACS) was held on 19th-20th October, welcoming 200 people online and in-person, from researchers to clinicians, neurologists, physiotherapists, medical
Reflecting on the 7th International ARSACS Symposium 2023 Read More »
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
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 »
Ataxia UK has a collaborative partnership with CureDRPLA, a US-based non-profit organization that funds scientific projects to advance research for the rare ataxia Dentatorubral-pallidoluysian atrophy (DRPLA). Both Dr Julie Greenfield
Sharing the success of the DRPLA Research Conference Read More »
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
Reflections on the Ataxia UK Annual Conference 2023 Read More »
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
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 »
The team at the Sheffield Ataxia Centre, led by Prof Marios Hadjivassiliou, has been investigating whether faulty genes called RFC1 seen in a condition called CANVAS (cerebellar ataxia, neuropathy and
On 12th September, Ataxia UK hosted its first networking event for UK-based researchers in London. We were excited to welcome almost 50 attendees, including researchers, clinicians, Ataxia UK representatives and
Ataxia UK hosts first-ever event for UK-based researchers 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 2023 Read More »
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 »
Dr Julie Greenfield, Ataxia UK’s Head of Research, interviewed by Junko Shiozawa, who is @curedrpla’s Advisory Board Member, and her daughter has DRPLA. Check out Junko’s channel: @CureDiseaseKuriChannel Camera
Interview with Junko Shiozawa 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 »
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
Results from PTC Therapeutics Friedreich’s ataxia trial Read More »
Late-onset spinocerebellar ataxia 27B (SCA27B) is newly discovered ataxia which is caused by a mutation in a gene called fibroblast growth factor 14 (FGF14). This discovery may lead to more
Newly discovered ataxia SCA27B shows promising response to a treatment 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. More specifically, CTI-1601
Preliminary results of Larimar Therapeutics FA trial show increase in frataxin levels 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 »
Ataxia UK were saddened to hear that Biogen have stopped their Phase I clinical trial for SCA3 therapeutic BIIB132 (MERA study). The trial was planned to be an international study,
Biogen announce they have stopped SCA3 clinical trial 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 »
