Research Impact Report 2024
The research that Ataxia UK funds has a real impact on the world of ataxia research, and our understanding of all ataxias. Every year we ask Ataxia UK-funded researchers to […]
Research Impact Report 2024 Read More »
PTC Therapeutics announces positive updates from their phase III trial of Vatiquinone in FA
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
In conversation with the London Ataxia Centre Speech and Language Therapist, Lucie Hogger
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
Intrabio announces FDA approval of their drug Levacetylleucine (Aqneursa) for the treatment of Niemann-Pick Disease Type C
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
Biohaven announces positive results in their study of Troriluzole for Spinocerebellar ataxia (SCA)
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. Â Â Those with SCA who
Satisfaction is significantly higher in those attending UK specialist ataxia centres compared with those who do not, new paper shows
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
In conversation with Dr. Federico Herrera part 2
Our Research Intern, Scarlett Parr-Reid, sits down with Dr. Federico Herrera (pictured below) for the second part of a two-part interview series, to discuss his team’s research project at the
In conversation with Dr. Federico Herrera part 2 Read More »
Update on genetic testing for SCA27B
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 »
Ataxia UK hosts Webinar on UK approval process for Skyclarys in adults with FA
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 announces positive results from their phase 1/2 clinical trial programme for FA cardiomyopathy
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
Ataxia UK hosts an educational webinar on the UK medicines approval process
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 »
In conversation with Dr Yi Shiau Ng, Consultant Neurologist, and Clinical Senior Lecturer at Newcastle University
Our research intern, Scarlett Parr-Reid, sits down with Dr Ng to discuss his research into mitochondrial disease and spinocerebellar ataxia type-6 (SCA6). Tell me a bit about yourself and your
In conversation with Dr. Federico Herrera, Group Leader of the Cell Structure and Dynamics (CSD) Lab from the Biosystems and Integrative Sciences Institute (BioISI) at the Faculty of Sciences of the University of Lisbon (Ciências ULisboa).
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
PROSPAX Consortium publishes two new research papers
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 »
In conversation with London Ataxia Centre Specialist Physiotherapist, Rachel Higgins
Rachel Higgins (BSc (Hons) MCSP) is a Clinical Specialist Neurological and Vestibular Physiotherapist at The National Hospital for Neurology and Neurosurgery (NHNN). She has worked within the field of neurology
In conversation with London Ataxia Centre Specialist Physiotherapist, Rachel Higgins Read More »
Larimar Therapeutics announces progress of the Nomlabofusp (CTI-1601) trial for Friedreich’s ataxia
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
In Conversation with London Ataxia Centre Consultant Neurologist, Professor Paola Giunti
Professor Paola Giunti is an award-winning Consultant Neurologist. She has specialised in ataxias for over 30 years, and set up the London Ataxia Centre in 2005. At the London Ataxia
Pre-implantation Genetic Testing licensed in UK for SCAR17
The Human Fertilisation and Embryology Authority (HFEA) is the UK’s independent regulator of fertility treatment and research using human embryos. They have licensed Pre-implantation Genetic Testing (PGT) to diagnose a
Pre-implantation Genetic Testing licensed in UK for SCAR17 Read More »
In conversation with London Ataxia Centre Specialist Ataxia Nurse, Suzanne Booth
Suzanne has worked as a neurology specialist nurse for 19 years, supporting people with a range of long-term neurological conditions. She has recently completed an MSc in Advanced Clinical Practice,
In conversation with London Ataxia Centre Specialist Ataxia Nurse, Suzanne Booth Read More »
Alterity Therapeutics presents data on the potential of ATH434 to treat FA
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 »
Study identifies gene that causes SCA4
A multinational research team led by University of Utah researchers has identified the gene that causes a rare type of late-onset ataxia called Spinocerebellar Ataxia Type-4 (SCA4). This discovery paves the
Study identifies gene that causes SCA4 Read More »
SBT-589 may be cardioprotective in FA
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 »
An interview with Netherlands-based PhD researcher Ilse Willemse, on the SPAX app for home monitoring in ARSACS and SPG7 as part of the European PROSPAX projectÂ
The PROSPAX (PROgression chart of SPAstic ataXias) project focuses on ARSACS and SPG7, and is a collaboration between neurologists in several sites across Europe and Canada. The overall aim is to gain
Voyager Therapeutics and Neurocrine Biosciences announce selection of a lead gene therapy candidate for their FA program
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
The PROSPAX Consortium publishes an infographic on their natural history study of individuals with ARSACS and SPG-7 in Europe
The PROSPAX (PROgression chart of SPAstic ataXias) project is a collaborative effort between neurologists across several sites throughout Europe and Canada. The aim is to better understand how spastic ataxias
Quality of care is higher in Specialist Ataxia Centres, European study finds
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 »
New study shows impacts of FA on daily life, wellbeing, and relationships in those with FA and their caregivers Â
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
Biohaven submits application to the EMA for a new drug approval of Troriluzole for SCA3Â
On October 26th 2023, the pharmaceutical company Biohaven submitted an application for a new drug approval of Troriluzole to the European Medicines Agency (EMA) for the treatment of Spinocerebellar Ataxia
Positive news regarding Skyclarys® approval in the EU
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 »
Paper published on study of effectiveness of omaveloxolone for Friedreich’s ataxia using natural history study data
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