Minoryx Therapeutics granted FDA Orphan Drug Designation in Friedreich’s Ataxia for new drug leriglitazone

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). Read more

Minoryx Therapeutics completes enrollment in FRAMES phase 2 trial with leriglitazone in Friedreich’s Ataxia

Recruitment of 39 patients in multicenter European phase 2 trial completed ahead of schedule Read more

Ataxia UK funds new trial investigating non-invasive brain stimulation as a potential therapy for all ataxias

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. Read more

Ataxia UK and FARA-funded project into heart disease associated with FA

Dr Carroll and his team have completed a project into a cause of heart disease associated with Friedreich’s ataxia (FA). Read more

Levels of the protein Nrf2 affect the development of the brain and spinal cord in Friedreich’s ataxia

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. Read more

The current state of biomarker research for Friedreich’s ataxia: a recent report from the 2018 FARA biomarker meeting

The US Friedreich’s ataxia charity FARA recently held a Biomarker Meeting reflecting on the current state of the development of these biomarkers. Read more

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