Toxic protein production reduced in SCA3 and SCA1 by genetic approach

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. Read more

Ataxia UK funds new FA research project developing new FA animal models to test gene therapy

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. Read more

BioMarin’s BMN 290 Programme: An Update for the Friedreich’s Ataxia Community

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. Read more

Survey seeking your advice on the treatment of sialorrhoea

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. Read more

The Neurological Alliance Report: Neuro Patience

In July 2019, The Neurological Alliance published the results of the 2018/19 National Neurology Patient Experience Survey. Read more

Gene-silencing treatment approved for hereditary amyloidosis

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. Read more

Multiple Sclerosis drug increases levels of frataxin

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. Read more

Correcting half the faulty heart muscle cells in mouse model of FA completely reverses cardiomyopathy

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. Read more

Global Ataxia Initiatives SCA Global and ARCA Global aim to advance ataxia research worldwide

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. Read more

Research Outcomes Report 2019

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. Read more

uniQure releases new data from Spinocerebellar Ataxia Type 3 gene therapy study

Leading gene therapy company uniQure have announced preclinical data on its gene therapy candidate, AMT-150, for the treatment of Spinocerebellar Ataxia 3 (SCA3). Read more

The eyes might be the windows to disease progression in FA

Professor Giunti and her team have identified a potential biomarker for FA by investigating the eye problems commonly found in patients. Read more

Mutation in RFC1 gene is likely to be common cause of late-onset ataxia

A mutation in gene found to be responsible for CANVAS, a specific form of late onset ataxia, is likely to also be responsible for forms of late onset ataxia with unknown causes. Read more

Real-time audio-biofeedback (ABF) shown to improve stability in degenerative cerebellar ataxia

A group of researchers from Germany have found that real-time audio bio-feedback improves stability in degenerative cerebellar ataxia. Read more

Preliminary evidence suggests that speech treatment could improve difficulty speaking in ARSACS

A recently published study has demonstrated that intensive home-based speech therapy can improve dysarthria (difficulty speaking) in autosomal recessive spastic ataxia Charlevoix-Saguenay (ARSACS). Read more

AMRC Impact Report

The AMRC Impact Report highlights the concrete ways that medical research charities make a difference to people and society through the research they fund Read more

New initiative for rare diseases launched by Finnish pharmaceutical company

The 'New Modalities Ecosystem’, launched in 2018 by the Finnish pharmaceutical company Orion, aims to exploit Finnish expertise to deliver new treatments and technologies for rare diseases. Read more

Effects of physiotherapy in patients with spinocerebellar ataxia type 7

A recent study investigating the effect of investigating the effect of physiotherapy on patients with spinocerebellar ataxia type 7 (SCA7) has found that it to significantly improve symptoms. Read more

Euro-ataxia annual meeting

Ataxia UK's Head of Research Julie Greenfield reports on the 2018 Euro-ataxia annual conference held in Frankfurt. Read more

EFACTS reports most common 'non-ataxia' symptoms in FA

The ongoing European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) study, part-funded by Ataxia UK has released a report evaluating the clinical variability in Friedreich’s ataxia (FA) symptoms. Read more