Dr Marta Olejniczak and her colleagues have used a genetic tool to silence the expression of mutated genes containing expanded CAG repeats in patient-derived cell lines of various ataxias.
Genetic approach to treat many spinocerebellar ataxias Read More »
Research shows exenatide increases frataxin in cell models and platelets in people with FA.
Exenatide in FA research Read More »
Exicure have announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development programme for their gene therapy technology.
Exicure to launch gene therapy programme in Friedreich’s ataxia Read More »
A research group in Italy will soon be launching a clinical trial in an effort to repurpose anti-viral drug Etravirine for use in FA.
Triplet Therapeutics are developing a new treatment that aims to address the underlying cause of the genetic dysfunction responsible for repeat expansion disorders including various SCAs.
Leading gene therapy uniQure have published data on their gene therapy candidate AMT-150.
uniQure publishes data from spinocerebellar ataxia type 3 gene therapy study Read More »
Chondrial Therapeutics recently announced that the first person has been treated in a Phase 1 clinical trial to assess the safety and tolerability of CTI-1601.
Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 FA trial Read More »
An Ataxia UK funded project using an innovative gene editing approach has identified 23 potential drug targets.
Friedreich’s ataxia research project identifies new drug targets Read More »
A project targeting the genetic cause of DRPLA, a rare form of ataxia, has received further funding from Ataxia UK.
Targeting the genetic cause of DRPLA Read More »
At the Euro-ataxia meeting in Frankfurt on 9th November 2018 a workshop was held to discuss the role of patients and patient groups in research. This led to the creation of the Euro-ataxia patient charter which has now been published.
Earlier this year the FDA granted an Orphan Drug Designation to an investigational treatment developed by Cadent Therapeutics for spinocerebellar ataxia. They plan to begin a clinical trial by the end of 2019.
Cadent Therapeutics aims to begin new trial in SCAs by the end of 2019 Read More »
A recent study has demonstrated depression has a significant impact on the quality of life of patients with Friedreich’s ataxia.
Depressive symptoms predict quality of life in FA patients and deserve recognition Read More »
Ataxia UK representatives attended the 3rd international ataxia research conference, organised by Ataxia UK, FARA, FARA Australia and GoFAR, took place on 14-16 November 2019.
The International Ataxia Research Conference 2019 in Washington DC was a great success Read More »
Minoryx Therapeutics has announced that its lead drug candidate, leriglitazone (MIN-102), has been given an Orphan Drug Designation for Friedreich’s Ataxia by the US Food and Drug Administration (FDA).
Reata Pharmaceuticals Inc have announced some brilliant news following a study into Friedreich’s ataxia treatment.
Very exciting results from Friedreich’s ataxia (FA) study Read More »
Recruitment of 39 patients in multicenter European
phase 2 trial completed ahead of schedule
An innovative brain stimulation technique known as ‘transcranial Direct Current Stimulation’ or ‘tCDS’ is being trialled as a potential therapy for all ataxias by an Ataxia UK funded research group in Italy.
Dr Carroll and his team have completed a project into a cause of heart disease associated with Friedreich’s ataxia (FA).
Ataxia UK and FARA-funded project into heart disease associated with FA Read More »
A recent study has shown that reduced Nrf2 levels in embryos taken from mice with FA affects the development of the brain and spinal cord.
The US Friedreich’s ataxia charity FARA recently held a Biomarker Meeting reflecting on the current state of the development of these biomarkers.
Spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) are caused by mutations in the Ataxin-3 and Ataxin-1 genes that. Researchers recently tested out a type of therapy that works by targeting these genes.
Toxic protein production reduced in SCA3 and SCA1 by genetic approach Read More »
Ataxia UK is funding a new project led by Dr Filip Lim’s research group that builds upon their previous research into a gene therapy for Friedreich’s ataxia.
We were disappointed to hear that on August 1, 2019 BioMarin announced plans to discontinue preclinical studies in the BMN 290 programme for Friedreich’s Ataxia.
BioMarin’s BMN 290 Programme: An Update for the Friedreich’s Ataxia Community Read More »
We are gathering information from people with ataxia to help the Scottish Medicines Consortium make an informed decision about a medicine used to treat excessive drooling as a symptom of a neurological condition.
Survey seeking your advice on the treatment of sialorrhoea Read More »
In July 2019, The Neurological Alliance published the results of the 2018/19 National Neurology Patient Experience Survey.
The Neurological Alliance Report: Neuro Patience Read More »
Gene-silencing has featured in the news lately, with the approval of a new type of medicine for the treatment of a condition known as hereditary amyloidosis.
Gene-silencing treatment approved for hereditary amyloidosis Read More »
In a recent study a team of researchers have shown that a drug used to treat Multiple Sclerosis (MS) is able to increase levels of frataxin.
Multiple Sclerosis drug increases levels of frataxin Read More »
A recent study has demonstrated that using gene therapy to correct half of the faulty heart muscle cells in a mouse model of Friedreich’s ataxia completely reverses cardiomyopathy.
Ataxia UK is on the steering committee of these two Global Ataxia Schemes, which hope to advance clinical research into ataxia on a worldwide scale.
Ataxia UK uses Researchfish to analyse the impact of Ataxia UK-funded research. This report summarises data from 39 grants, awarded between 2009 and 2018.
Research Outcomes Report 2019 Read More »
