Reata Pharmaceuticals Inc have announced some brilliant news following a study into Friedreich's ataxia treatment. 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
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
Dr Carroll and his team have completed a project into a cause of heart disease associated with Friedreich’s ataxia (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. Read more
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
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 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
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
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
In July 2019, The Neurological Alliance published the results of the 2018/19 National Neurology Patient Experience Survey. 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. Read more