September 21, 2022

Results of Phase 3 clinical trial of tofersen for MND

Around 5,000 people in the UK have motor neurone disease (MND) – also known as amyotrophic lateral sclerosis (ALS). Two percent of MND cases are caused by a faulty gene called SOD1.

Researchers from the University of Sheffield Institute for Translational Neuroscience (SITraN) and the Sheffield NIHR Biomedical Research Centre now have the results of their phase 3 clinical trial looking at use of the investigational drug tofersen in 108 MND patients known to have the faulty SOD1 gene.

Results of the trial have been published in the New England Journal of Medicine.

Journalists dialled in to this briefing to hear from the scientists who are running the trial about their results.

Speakers included:

Professor Dame Pamela Shaw, Professor of Neurology and Director of Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield

Professor Chris McDermott, Professor of Translational Neurology at Sheffield Institute for Translational Neuroscience (SITraN), University of Sheffield

This Briefing was accompanied by an SMC Roundup of Comments.