A landmark trial supported by NIHR researchers has found an experimental drug targeting a rare form of motor neurone disease showed significant physical benefits after 12 months.
The phase 3 clinical trial, published in the New England Journal of Medicine, has shown that the new investigational drug tofersen can slow and reduce progression of the disease in patients with MND caused by the faulty SOD1 gene.
MND is a disorder that affects the nerves – or motor neurones – in the brain and spinal cord that form the connection between the nervous system and muscles, enabling movement of the body. In people with MND, the messages from these nerves gradually stop reaching the muscles, leading them to weaken, stiffen and eventually waste.
Read more at: https://www.nihr.ac.uk/news/promising-motor-neurone-disease-drug-helps-slow-disease-progression/31540