On 25th September 2024, the pharmaceutical company Intrabio announced that the FDA in the US has approved their small molecule drug Levacetylleucine for the treatment of Niemann-Pick Disease Type C […]
On September 23rd, 2024, the pharmaceutical company Biohaven announced positive topline results from their study of the drug Troriluzole to treat SCAs 1,2,3,6,7,8 and 10. Those with SCA who
Many people with ataxia do not have a specific genetic diagnosis and the cause of their ataxia is unknown. In 2023, two papers were published which identified a genetic mutation
Update on genetic testing for SCA27B 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
Our research intern, Scarlett Parr-Reid, sits down with Dr. Herrera to discuss his research on spastic ataxia type-8 (SPAX8). Can you share a little about yourself, your background as a
The PROSPAX Consortium has recently published two new research papers as part of the PROSPAX project, an international project that aims to better understand how spastic ataxias progress over time
PROSPAX Consortium publishes two new research papers Read More »
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 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 of
Quality of care is higher in Specialist Ataxia Centres, European study finds 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
