On 4th June 2025, the Biotechnology company Design Therapeutics announced that they have dosed the first person with Friedreich’s ataxia (FA) in their phase 1 clinical trial of their drug […]
Design Therapeutics RESTORE-FA trial updates page Read More »
Lexeo Therapeutics is a genetic medicine company, developing treatments for a number of conditions including cardiomyopathy associated with Friedreich’s ataxia (FA). Cardiomyopathy is a heart condition involving stretching, thickening, or stiffening of the heart muscle, making it harder to
Lexeo Therapeutics update page Read More »
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 »
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
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
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 2024, 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
Updates on the Voyager Therapeutics and Neurocrine Biosciences FA programme Read More »
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 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 »
