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 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 …
Larimar Therapeutics announces progress of the CTI-1601 trial for Friedreich’s ataxia Read More »
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 »
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 …
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 »
Article written by Dr Silvia Prades – DRPLA Research Manager On February 22nd, I organised a meet up for families affected by DRPLA who are based in Wales. I travelled …
Successful meet up in Cardiff with DRPLA families 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 »
