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 December 2025 the government announced that from April 2026 it will increase the standard NICE cost-effectiveness thresholds, which are used when evaluating new medicines for use in the NHS, from £20,000 – £30,000 to £25,000 – £35,000. Notably, the threshold has
Changes to the NICE cost-effectiveness threshold Read More »
NHS England opened a consultation to seek feedback on an operational framework they are developing to support the future use of individualised genetic therapies in the NHS in England. They
Existing medicines regulatory processes do not consider the challenges that accompany treatment development for rare diseases. The regulatory body for medicines in the UK, the MHRA, has recognised this and is planning a major change by proposing a new framework for rare diseases. We welcome their report,
MHRA publishes plans to reform medicines regulatory systems for rare diseases Read More »
This study, based at the Institute of Neurology, University College London, is led by Neuroscientist Dr Tim West, and funded by the Royal Academy of Engineering and the Rosetrees Trust.
A new project studying neurostimulation in ataxia Read More »
Last month, on 7th November, the Ataxia UK-accredited Sheffield Ataxia Centre hosted an ataxia study day for healthcare professionals at the University of Sheffield, chaired by Consultant Neurologists Professor Marios Hadjivassiliou and Dr Priya Shanmugarajah, and organised
The Sheffield Ataxia Centre’s 2025 ataxia study day for healthcare professionals Read More »
