24/10/2022
A phase 3 Clinical trial of the investigational drug tofersen in patients with MND Caused by the faulty SOD1 gene, has shown it can slow and reduce progression of the disease. Patients on the trial reported better patient mobility and lung function after 12 months. Researchers form the University of Sheffield Institute of Translational Neuroscience and the NIHR Sheffield Biomedical Research Center found that, though biomarkers in patients cerebrospinal fluid showed improvement at six months, it took 12 months for identification of physical benefits.
108 Patients took part in the clinical trial, funded by the biotechnology company Biogen Inc. Sheffield was the major trial site in UK. Around 5000 people in the UK have MND also known as amyotrophic lateral sclerosis, with two percent developing MND due to a faulty gene called SOD1.