New research that stemmed from a collaboration between the London Ataxia Centre and the Children’s Hospital of Philadelphia has identified the raise of a protein called NfL, as a potential […]
NfL may be a potential biomarker in Friedreich’s ataxia 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 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 »
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 »
