Exenatide in FA research

Research shows exenatide increases frataxin in cell models and platelets in people with FA. Read more

Exicure to launch gene therapy programme in Friedreich’s ataxia

Exicure have announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development programme for their gene therapy technology. Read more

New clinical trial testing HIV treatment, Etravirine, as potential therapy for FA to begin this year in Italy

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. Read more

uniQure publishes data from spinocerebellar ataxia type 3 gene therapy study

Leading gene therapy uniQure have published data on their gene therapy candidate AMT-150. Read more

Triplet Therapeutics is developing a fundamentally different treatment for repeat expansion disorders such as various spinocerebellar ataxias

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. Read more

Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 FA trial

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. Read more

Friedreich’s ataxia research project identifies new drug targets

An Ataxia UK funded project using an innovative gene editing approach has identified 23 potential drug targets. Read more

Targeting the genetic cause of DRPLA

A project targeting the genetic cause of DRPLA, a rare form of ataxia, has received further funding from Ataxia UK. Read more

Euro-ataxia publishes patient charter outlining mission for patient involvement in ataxia clinical trials

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. Read more

The International Ataxia Research Conference 2019 in Washington DC was a great success

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. Read more

Depressive symptoms predict quality of life in FA patients and deserve recognition

A recent study has demonstrated depression has a significant impact on the quality of life of patients with Friedreich’s ataxia. Read more

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