Following clearance by the US drug regulatory body the Food and Drug Administration (FDA) in July 2023, the pharmaceutical company Larimar Therapeutics has completed full enrolment and dosing of the …
Larimar Therapeutics announces progress of the CTI-1601 trial for Friedreich’s ataxia Read More »
Following results from their MOVE-FA phase 3 clinical trial of the drug vatiquinone in children and adults with Friedreich’s Ataxia (FA), the pharmaceutical company PTC Therapeutics wrote to the US …
Update from PTC Therapeutics on their MOVE-FA programme for Friedreich’s ataxia Read More »
On Friday 20th and Saturday 21st October 2023, Ataxia UK held their first ever hybrid in-person and virtual annual conference for families affected by ataxia. The conference, which took place …
Reflections on the Ataxia UK Annual Conference 2023 Read More »
Design Therapeutics is Biotechnology company developing DT-216, which is a potential treatment for Friedreich’s ataxia (FA). FA is caused by a change to the frataxin gene. Genes are made …
Design Therapeutics announce results from Phase 1 FA trial Read More »
Larimar Therapeutics is a biotechnology company developing treatments for rare conditions, including Friedreich’s ataxia (FA). They are testing a treatment for FA called CTI-1601, which is a protein replacement therapy …
Larimar Therapeutics receives FDA clearance to proceed with next stage of trial Read More »
Lexeo Therapeutics is a company developing gene therapy for people with FA cardiomyopathy. They recently announced the completion of the first cohort in their Phase I/II trial, testing their gene …
PTC Therapeutics have announced the initial results of their Phase 3 MOVE-FA trial of vatiquinone (PTC-743) in people with FA. Vatiquinone targets an enzyme called 15-lipoxygenase, which is a regulator …
Results from PTC Therapeutics Friedreich’s ataxia trial Read More »
Larimar Therapeutics is a biotechnology company, developing a treatment for Friedreich’s ataxia (FA) called CTI-1601. CTI-1601 replaces frataxin, which is the protein that is reduced in FA. More specifically, CTI-1601 …
Preliminary results of Larimar Therapeutics FA trial show increase in frataxin levels Read More »
Larimar Therapeutics is a biotechnology company, developing a treatment for Friedreich’s ataxia (FA) called CTI-1601. CTI-1601 replaces frataxin, which is the protein that is reduced in FA. Larimar Therapeutics recently …
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 »
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 …
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, …
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 …
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 …
An update about Reata Pharmaceuticals Omaveloxolone trial in FA
Exicure have announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development programme for their gene therapy technology.
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.
An Ataxia UK funded project using an innovative gene editing approach has identified 23 potential drug targets.
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.
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.
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.
Omaveloxolone has recently been given an Orphan drug designation for Friedreich’s ataxia the European Medicines Agency.
A new placebo-controlled trail in humans, testing a novel therapy, Tak-831 for Friedreich’s ataxia, has begun in America.
