Exicure have announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development programme for their gene therapy technology.
Exicure to launch gene therapy programme in Friedreich’s ataxia 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.
Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 FA trial Read More »
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 »
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
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 »
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.
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 »
Omaveloxolone has recently been given an Orphan drug designation for Friedreich’s ataxia the European Medicines Agency.
New EU Orphan drug designation for Freidreich’s ataxia Read More »
A new placebo-controlled trail in humans, testing a novel therapy, Tak-831 for Friedreich’s ataxia, has begun in America.
Results of a study evaluating TAK-831 in adults with Friedreich’s ataxia (FA) 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 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).
The latest findings from a trial testing a potential new gene therapy for Friedreich’s ataxia have been announced.
Data released from preclinical study into gene therapy for Friedreich’s ataxia Read More »
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