Experimental motor neurone disease therapy shows early promise
An experimental therapy developed to address the genetic cause of a Amyotrophic lateral sclerosis (ALS), rare form of motor neurone disease (MND) has shown encouraging results in early research supported by the NIHR.
The phase 1-2 study, supported by the National Institute for Health Research and published in the New England Journal of Medicine, has shown tofersen is safe and well tolerated in people with a genetic form of MND.
Around 5,000 people in the UK have MND, a disorder that affects the nerves in the brain and spinal cord that form the connection between the nervous system and muscles, enabling movement of the body.
In people with the disease, the messages from these nerves gradually stop reaching the muscles, leading them to weaken, stiffen and eventually waste, affecting a patient’s ability to walk, talk, use their arms and hands, eat and breathe.
Read more: http://www.pharmatimes.com/news/experimental_motor_neurone_disease_therapy_shows_early_promise_1344775
The phase 1-2 study, supported by the National Institute for Health Research and published in the New England Journal of Medicine, has shown tofersen is safe and well tolerated in people with a genetic form of MND.
Around 5,000 people in the UK have MND, a disorder that affects the nerves in the brain and spinal cord that form the connection between the nervous system and muscles, enabling movement of the body.
In people with the disease, the messages from these nerves gradually stop reaching the muscles, leading them to weaken, stiffen and eventually waste, affecting a patient’s ability to walk, talk, use their arms and hands, eat and breathe.
Read more: http://www.pharmatimes.com/news/experimental_motor_neurone_disease_therapy_shows_early_promise_1344775
