On October 27th-28th 2025, representatives from Ataxia UK attended the first Clinical Trials in Ataxia (CTAX) conference in Amsterdam. CTAX was organised by Ataxia UK, the National Ataxia Foundation […]
Reflections on the 2025 Clinical Trials in Ataxias Conference Read More »
The Human Fertilisation and Embryology Authority (HFEA), the UK’s independent regulator of fertility treatment and research using human embryos, has licensed Pre-implantation Genetic Testing (PGT) to diagnose a rare form of
Pre-implantation genetic testing approved to diagnose SCAN3 Read More »
In September 2024 Biohaven announced positive results from their study of troriluzole to treat SCA 1, 2, 3, 6, 7, 8 and 10. Following these results they submitted a new
FDA does not approve new drug application for troriluzole for spinocerebellar ataxias Read More »
The pharmaceutical company Lexeo Therapeutics has announced positive updates from their phase I/II trial of the gene therapy LX2006 to treat cardiomyopathy in Friedreich’s ataxia (FA). Interim data has shown
Lexeo shares positive data in trial of LX2006 for FA cardiomyopathy Read More »
As part of the Ataxia UK 2025 virtual annual conference, Emma Foster, Clinical Nurse Specialist at the Sheffield Ataxia Centre (pictured to the left) gave a talk on managing sleep
Managing sleep and fatigue symptoms in ataxias Read More »
On 29th September 2025, the pharmaceutical company Larimar Therapeutics announced positive results from their phase II trial of the drug Nomlabofusp for FA. Nomlabofusp is designed to increase levels of
The pharmaceutical company Intrabio, who developed the recently FDA-approved drug AQNEURSA for people with Niemann-Pick Disease Type C, has announced their plans to begin a phase III trial of the
Intrabio to conduct phase III trial in SCA6 and Episodic Ataxia Type-2 Read More »
Current clinical rating scales to measure disease progression in people with spasticity and ataxia are focused solely on either spasticity symptoms (stiffness of muscles leading to involuntary spasms) or ataxia
Ataxia UK funds research that aims to find treatments and cures for the ataxias. This would not be possible without your continued generosity in donating to support this mission. Thank
Celebrating the impact of Ataxia UK-funded research Read More »
The pharmaceutical company Arrowhead Therapeutics has begun recruitment for their phase 1 randomised placebo-controlled trial of ARO-ATXN2 in adults with SCA2. The programme is a partnership with the pharmaceutical company
Arrowhead Pharmaceuticals to open UK sites in SCA2 trial Read More »
Prof Anja Lowit, Speech and Language Therapist based Strathclyde University, Glasgow, together with Prof Marios Hadjivassiliou from the Sheffield Ataxia Centre have published a new research paper looking at a
On 19th August 2025, the pharmaceutical company PTC Therapeutics announced the disappointing news that the US drug regulators the FDA have not approved the drug Vatiquinone to treat adults and
Upcoming project: Co-designing audio support messages with and for people with ataxia: a telephone version of the “I’m not drunk” ID card Principal Investigators: Dr Maria Cairney and Prof Anja
Upcoming project: Subtyping Hereditary Cerebellar Ataxias based on multimodal disease progression patterns Subtyping Hereditary Cerebellar Ataxias based on multimodal disease progression patterns Principal investigators: Dr Susmita Saha, Prof Nellie Georgiou-Karistianis,
Upcoming project: Investigating The Molecular Mechanisms of SCA2 Using 3D Cerebellar Organoids and Multi-Omics Approaches: A Platform for Advancing Ataxia Research Principal applicants: Prof Jorgen Erik Nielsen, Senior Researcher Patrick
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 »
Title: Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia Principal researchers: Boubker Zaaimi (Aston University, UK), Prof Stuart Baker (Newcastle University, UK), Prof Mark Baker (Newcastle University, UK)
Augmented Reality-Based Closed-Loop Rehabilitation for Postural Instability in Ataxia Read More »
A recent paper published in the scientific journal Cell Reports Medicine has shown that a type of non-invasive brain stimulation called transcranial alternating current stimulation (tACS) provides benefits to people
Transcranial brain stimulation can improve symptoms in SCA3 Read More »
Ataxia UK has funded a number of research projects in Friedreich’s ataxia (FA) by Dr Sara Anjomani-Virmouni, a researcher based at Brunel University, London. As a result of this funded
Antioxidants as a potential therapy for Friedreich’s ataxia Read More »
Upcoming Project: Caring for people with progressive ataxia – lived experience of family carers and co-development of support mechanisms Principal researchers: Prof Anja Lowit and Prof Christopher Graham, Strathclyde
On 4th June, 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 DT-216P2.
First FA patient dosed in RESTORE-FA trial Read More »
On Friday 23rd May, Ataxia UK hosted a webinar and Q&A on access to omaveloxolone for adults with Friedreich’s ataxia (FA) in the UK. This webinar was presented in partnership
We recently shared with the community the disappointing news about that Biogen have withdrawn their application to NICE (the body that makes decisions on reimbursement of medicines in the UK)
As FA progresses, I am getting more and more fearful about trying to go out for short walks. I know I am putting on weight. I bought a static bike/cross-trainer
Living with Late Onset – Exercise and FA Read More »
On 7th and 8th May, Ataxia UK’s Research Communications Officer, Scarlett Parr-Reid, attended Beacon for Rare Diseases and Remedi4All’s second International Drug Repurposing Conference (IDR25), held in Amsterdam. Drug repurposing
Reflections from the International Drug Repurposing Conference 2025 Read More »
In March, Biohaven shared that they have taken the decision to withdraw their new drug application to the European Medicines Agency (EMA) for Troriluzole to treat adults with all Spinocerebellar
Biohaven withdraws Troriluzole application to EMA for Spinocerebellar ataxias Read More »
Biogen have shared their plan to initiate their BRAVE study, a global phase 3 trial of Omaveloxolone (SKYCLARYS®) in children aged 2-15 years. The trial will study the effects and
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 »
Two years ago, at the age of 44, I was diagnosed with a rare condition called Friedreich’s Ataxia (FA). I had balance and speech symptoms for a few years and
Living with Late Onset – Clare’s Friedreich’s ataxia Story Read More »
A study led by Dr Sarah Milne at the Murdoch Children’s Research Institute, Australia, has recently been published in the journal ANNALS of Neurology. The study looked at the impact
