Biogen have shared their plan to initiate their BRAVE study, a global phase 3 trial of Omaveloxolone (SKYCLARYS®) in children aged 2-15 years. The trial will study the effects and […]
The UK medicines regulator (MHRA) approves the first treatment for Friedreich’s ataxia (FA), Omaveloxolone (Skyclarys). On 23 April 2025, the MHRA approved Omaveloxolone for the treatment of those aged 16
MHRA approves Omaveloxolone for Friedreich’s ataxia Read More »
On March 24th 2025, the pharmaceutical company Lexeo Therapeutics provided a number of updates on their phase 1/2 trial of the gene therapy drug LX2006 for Friedreich’s ataxia (FA) cardiomyopathy.Â
On March 24th 2025, the pharmaceutical company Larimar Therapeutics announced their plans to start conducting a phase 3 trial of their Friedreich’s ataxia (FA) drug Nomlabofusp in mid-2025. This follows
Larimar Therapeutics announce plans for phase 3 trial of Nomlabofusp for FA Read More »
The International Congress for Ataxia Research (ICAR) 2024 took place in London from 12th – 15th November. It was organised by Ataxia UK, the National Ataxia Foundation, the Friedreich’s Ataxia
Highlights of the International Congress for Ataxia Research 2024 Read More »
On 8th October 2024, the pharmaceutical company PTC Therapeutics announced positive results from their phase III trial of their drug Vatiquinone for adults and children with Friedreich’s ataxia (FA). Analysis
On 23rd July, Ataxia UK hosted a webinar and Q&A on the UK approval process for Omaveloxolone (Skyclarys) in adults with FA. This was in partnership with the consultancy Realise
Ataxia UK hosts Webinar on UK approval process for Skyclarys in adults with FA Read More »
Lexeo Therapeutics is a pharmaceutical company developing a gene therapy called LX2006 for people with Friedreich’s ataxia (FA) hypertrophic cardiomyopathy (disease of the heart involving stretching, thickening, or stiffening of
On 12th July Ataxia UK hosted a webinar on how medicines get approved for use in the UK and how decisions are made on their funding in the NHS. The
Ataxia UK hosts an educational webinar on the UK medicines approval process Read More »
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
The biotechnology company Alterity Therapeutics presented data from their lead Friedreich’s ataxia (FA) drug candidate ATH434 at the 2024 US World Orphan Drug Congress. In FA, a genetic change in
Alterity Therapeutics presents data on the potential of ATH434 to treat FA Read More »
On March 19th, the pharmaceutical company Stealth BioTherapeutics presented data on their molecule called SBT-589 in preclinical models of Friedreich’s ataxia (FA) at the Wellcome Trust Conference on Mitochondrial Medicine-Therapeutic
SBT-589 may be cardioprotective in FA Read More »
On 26th February, the biotechnology company Voyager Therapeutics announced that as part of their collaboration with the biopharmaceutical company Neurocrine Biosciences, they have selected a lead development candidate in their
Ataxia UK is delighted with today’s announcement from the EMA’s Committee for Medicinal Products for Human Use (CHMP), the regulatory body that makes recommendations on drug approvals in the EU.
Positive news regarding Skyclarys® approval in the EU Read More »
The natural history of Friedreich’s ataxia (FA) has been studied as part of a project called the Friedreich’s ataxia Clinical Outcome Measures Study (FACOMS). Natural history studies track the course
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
Lexeo Therapeutics FA trial update Read More »
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
Larimar Therapeutics – FA trial update Read More »
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
