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 »
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
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
Research by Prof Anja Lowit and her team at Strathclyde University, Glasgow, studying the effectiveness of a new type of speech therapy for some progressive ataxias, has shown some promising
