An Ataxia UK funded project using an innovative gene editing approach has identified 23 potential drug targets.
Friedreich’s ataxia research project identifies new drug targets 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.
A recent study has demonstrated depression has a significant impact on the quality of life of patients with Friedreich’s ataxia.
Depressive symptoms predict quality of life in FA patients and deserve recognition 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).
Reata Pharmaceuticals Inc have announced some brilliant news following a study into Friedreich’s ataxia treatment.
Very exciting results from Friedreich’s ataxia (FA) study Read More »
Recruitment of 39 patients in multicenter European
phase 2 trial completed ahead of schedule
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.
Dr Carroll and his team have completed a project into a cause of heart disease associated with Friedreich’s ataxia (FA).
Ataxia UK and FARA-funded project into heart disease associated with 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.
The US Friedreich’s ataxia charity FARA recently held a Biomarker Meeting reflecting on the current state of the development of these biomarkers.
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.
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.
BioMarin’s BMN 290 Programme: An Update for the Friedreich’s Ataxia Community Read More »
In July 2019, The Neurological Alliance published the results of the 2018/19 National Neurology Patient Experience Survey.
The Neurological Alliance Report: Neuro Patience Read More »
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.
Multiple Sclerosis drug increases levels of frataxin Read More »
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.
Professor Giunti and her team have identified a potential biomarker for FA by investigating the eye problems commonly found in patients.
The eyes might be the windows to disease progression in FA Read More »
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.
EFACTS reports most common ‘non-ataxia’ symptoms in FA Read More »
Ataxia UK’s Head of Research Julie Greenfield reports on the 2018 Euro-ataxia annual conference held in Frankfurt.
Euro-ataxia annual meeting Read More »
Ataxia UK has been talking with Minoryx Therapeutics, a company focusing on developing new drugs for rare conditions, and we are delighted with this new trial being announced.
Experiments in the laboratory using skin cells taken from people with Friedreich’s ataxia have shown that these cells may be more susceptible to damage caused by certain sun rays.
Friedreich’s ataxia may cause greater susceptibility to skin damage by UVA rays Read More »
We are delighted that on 20 February 2019 the Ataxia UK Medical Guidelines were published in the Orphanet Journal of Rare Diseases.
Ataxia UK medical guidelines published in peer-reviewed journal Read More »
A study has found that a rapid, non-invasive eye exam could potentially be used as a tool for assessing Friedreich’s ataxia patients and as a biomarker in clinical trials.
Non-invasive eye exam found to predict disease severity in Friedreich’s ataxia Read More »
Pharmaceutical company, Reata, has announced new preclinical data that supports the potential use of omaveloxolone in Friedreich’s ataxia.
Positive results announced for potential FA treatment Read More »
Studies have shown that the frataxin gene may be activated by a wide variety of synthetic antisense oligonucleotides
New options for targeting gene mutation in FA described in nucleic acid therapeutics Read More »
Results from a phase 2 clinical trial suggest that a new therapy may improve neurological function and prevent disease progression relative to the natural course of Friedreich’s ataxia (FA).
Euro-ataxia held the latest annual meeting on 9-10th November 2018 in Frankfurt, Germany, hosted by the German ataxia charity DHAG, organised by Ataxia UK and sponsored by BioMarin.
Euro-ataxia conference 2018 Read More »
Results from MOXIe study reveal potential therapy for Friedreich’s ataxia, omaveloxolone, is well tolerated.
Results published from part 1 of Friedreich’s ataxia drug trial Read More »
Results from a study conducted at the London Ataxia Centre have shown that 3 proteins which may represent new biomarkers in Friedreich’s ataxia (FA).
Study discovers 3 potential blood based biomarkers for FA Read More »
NHS England has included hereditary ataxia in the final draft of the National Genomic Test Directory for rare and inherited disorders and cancer
Final draft of National Genomic Test Directory includes hereditary ataxia Read More »
Study finds anti-viral drug leads to decrease in frataxin levels and may be a potential future candidate for treating Friedreich’s ataxia (FA).
HIV treatment identified as potential future therapy for FA Read More »
