Design Therapeutics have announced positive data from the first part of their Phase I clinical trial of DT-216. They found that treatment with DT-216 increased frataxin mRNA. Frataxin mRNA is […]
Design Therapeutics announce positive data from Friedreich’s ataxia trial Read More »
LEXEO Therapeutics is a gene therapy company, developing treatments for a number of conditions including cardiomyopathy associated with FA. In February 2022, LEXEO Therapeutics announced that the FDA had cleared
LEXEO Therapeutics announce plans for clinical trial Read More »
Please see below an update from Novartis Gene Therapies on their Friedreich’s ataxia program: February 2022 Novartis Gene Therapies had been developing a therapeutic approach for Friedreich’s ataxia. One candidate,
Update on Novartis Gene Therapies Friedreich’s ataxia Program Read More »
The US biopharmaceutical company Stealth Biotherapeutics is running a phase 1/2 trial of Elamipretide for the treatment of vision loss and cardiac disease in Friedreich’s ataxia (FA). The trial started
Stealth Biotherapeutics begins phase 1/2 trial of Elamipretide in FA Read More »
Update 27/09/2022 In May 2021, the US Food and Drug Administration (FDA) put a full clinical hold on Larimar Therapeutics programme for Friedreich’s ataxia (FA) drug CTI-1601. CTI-1601 is a
Larimar Therapeutics: FA Drug Update Read More »
Update 17/11/2022 Omav data presented at the International Congress for Ataxia Research (ICAR 2022 – Dallas, Texas) Dr David Lynch, from the University of Pennsylvania and Children’s Hospital of Philadelphia,
Reata Pharmaceuticals – Omaveloxolone Update Read More »
Ataxia UK, along with Ataxia Global Initiative and National Ataxia Foundation, recently partnered with Critical Path Institute (C-Path) for the launch of their Critical Path to Therapeutics for the Ataxias
FARA’s campaign asks people affected by Friedreich’s ataxia to sign their open letter. The letter addresses the US regulator, FDA, and Reata pharmaceuticals. The letter requests Reata to submit a
Open letter to Reata and the FDA Read More »
Recently, Ataxia UK had a meeting with representatives from Larimar Therapeutics, a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, to discuss possible collaborations to help progress
FA drug clinical trial update: Larimar Therapeutics Read More »
In October 2019, Reata Pharmaceuticals announced positive top-line results from their MOXIe trial testing Omaveloxolone (or Omav) in Friedreich’s ataxia (FA). In the MOXIe trial, Reata used the modified Friedreich’s
Omaveloxolone (Omav) Update Read More »
An update about Reata Pharmaceuticals Omaveloxolone trial in FA
Disappointing news from Reata and FDA regarding Omaveloxolone 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.
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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