Reata Pharmaceuticals Inc have announced some brilliant news following a study into Friedreich's ataxia treatment. Read more
Read more
Researchers in Japan have shown that a compound called Arginine is able to prevent harmful protein clumps from forming in cell models of polyglutamine disorders. Read more
A recent study led by a group of researchers in Portugal tested the effect of trehalose on an animal model of SCA3. Read more
Read a summary of the impact of Ataxia UK-funded research in the Research Impact Report 2020 Read more
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. Read more
Research shows exenatide increases frataxin in cell models and platelets in people with FA. 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. 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. Read more
Leading gene therapy uniQure have published data on their gene therapy candidate AMT-150. Read more
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 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
An Ataxia UK funded project using an innovative gene editing approach has identified 23 potential drug targets. Read more
A project targeting the genetic cause of DRPLA, a rare form of ataxia, has received further funding from Ataxia UK. 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. Read more
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. 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. Read more
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 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
Recruitment of 39 patients in multicenter European phase 2 trial completed ahead of schedule Read more