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 »
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 »
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 »
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 …
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 »
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 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 …
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 »
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 …
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 »
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 …
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 »
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 »
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 …
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 »
In January 2022, researchers at UCL/UCLH, Children’s Hospital of Philadelphia, Ataxia UK and Friedreich’s Ataxia Research Alliance (FARA), published a paper on the attitudes of people with ataxia towards clinical …
Paper published on the attitudes of people with ataxia towards clinical trials Read More »
Please see below an update from Novartis Gene Therapies on their Friedreich’s ataxia program: February 2022 Novartis Gene Therapies had been developing a therapeutic approach for Friedreich’s ataxia. One candidate, …
Update on Novartis Gene Therapies Friedreich’s ataxia Program Read More »
An article published in the Lancet Neurology showed that whole genome sequencing (WGS) can detect common inherited neurological disorders, including some ataxias. Genetic testing can be a slow process, with …
A simple DNA test can detect common neurological disorders Read More »
The Ataxia Magazine Issue 215 reported on a new paper on Friedreich’s ataxia (FA) from the team led by Professor Paola Giunti at the London Ataxia Centre, which has been …
In the clinic, assessment of cerebellar ataxias has been largely confined to clinical rating scales (e.g. Scale for Assessment and Rating of Ataxia -SARA-), gait laboratories, imaging of the brain, …
Development and Validation of a Patient-Reported Questionnaire of Ataxia Progression Read More »
Update 27/09/2022 In May 2021, the US Food and Drug Administration (FDA) put a full clinical hold on Larimar Therapeutics programme for Friedreich’s ataxia (FA) drug CTI-1601. CTI-1601 is a …
Update 17/11/2022 Omav data presented at the International Congress for Ataxia Research (ICAR 2022 – Dallas, Texas) Dr David Lynch, from the University of Pennsylvania and Children’s Hospital of Philadelphia, …
