On Wednesday 5th February, Ataxia UK hosted a webinar and Q&A as part of Wellbeing Week, a week from 3rd – 7th February dedicated to providing support and information to […]
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
Recently, our research intern Scarlett Parr-Reid sat down with Lucie Hogger, Speech and Language Therapist at the London Ataxia Centre to talk about the speech and language therapy services offered
Ataxia UK has worked closely with the London Ataxia Centre researchers and other experts on a research study assessing the impact of specialist ataxia centres in Europe. This was part
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
Findings from a recent study, ‘Health-related quality of life in Friedreich Ataxia: A study of impacts on patients and caregivers’, were presented at the International Society for Pharmacoeconomics and Outcomes
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
Research by Prof Anja Lowit and her team at Strathclyde University, Glasgow, studying the effectiveness of a new type of speech therapy for some progressive ataxias, has shown some promising
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 »
In this article, Owen Gittins, a 3rd year undergraduate student in biochemistry at the University of Cambridge, shares his journey into and involvement in Friedreich’s ataxia (FA) research. My
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 »
