Spinocerebellar ataxias (SCAs) are a group of rare, progressive hereditary genetic disorders that affects the cerebellum, brain stem and spinal cord. Currently, more than 30 types of SCAs have been identified. SCAs are caused by genetic mutations, which lead to faulty […]
VICO Therapeutics Clinical Trial updates Read More »
In 2021, Ataxia UK co-funded a research project with the National Ataxia Foundation, ‘Assessment of ataxia severity under real-life conditions with SARAhome: A multicenter study in spinocerebellar ataxia type 3 (SCA3)’. This project was led by Dr
New paper published on the SARAhome scale to remotely assess ataxia symptoms in SCA3 Read More »
Spinocerebellar ataxia type-3 (SCA3) is one of the most common types of SCA worldwide, and is characterised by progressive decline in daily function and quality of life. Whilst cerebellar ataxia
In a project funded by Ataxia UK, Dr Andreia Teixeira-Castro (pictured left) and her team at the University of Minho, Portugal, have investigated the therapeutic potential of the anti-depressant vortioxetine
Sharing results from Ataxia UK-funded project looking at potential treatment for SCA3 Read More »
This project, led by Prof Karen Anthony at the University of Northampton and Dr Ronald Buijsen at Leiden University Medical Centre, aimed to evaluate the potential of targeting RNA to
Ataxia UK-funded project shows potential for SMaRT gene therapy to treat SCA1 Read More »
Dr Sandra Macedo-Ribiero of the University of Porto, Portugal, has shared results from her project co-funded by Ataxia UK, ‘NanoSCA3: Development of brain-targeted nanobodies for application in spinocerebellar ataxia type
Promising results from Ataxia UK co-funded project targeting SCA3 using nanobodies Read More »
Researchers Prof Bart van de Warrenburg, Dr Roderick Maas and Kristofoor Leeuwenberg at Radboud University Medical Center in the Netherlands have recently published a paper in the journal Cerebellum on
New paper published on measuring treatment effectiveness in people with SCA3 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 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 »
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 »
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 »
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 »
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
On 1st December 2023, the pharmaceutical company Seelos Therapeutics announced they had stopped their phase 2/3 programme known as STRIDES, which had been investigating a new drug for Spinocerebellar Ataxia
Seelos Therapeutics has stopped their phase 2/3 trial of Trehalose for SCA3 Read More »
