Scientists hope a new drug could help slow motor neuron disease

Scientists involved in clinical trials say they have found a “promising” new drug that could slow the progression of the disease Motor neuron disease (MND).

In the global studies, some patients with a faulty SOD1 gene reported that they had better lung and mobility function a year after taking the drug Tofersen.

Tofersen is an investigational drug, which means that scientists analyzed its effectiveness and safety during the studies.

The findings were published in the New England Journal of Medicine, and scientists said they were “remarkable” for a illness characterized by “relentless decline”.

Dame Pamela Shaw, Professor of Neurology and Director of the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield, said: “I have more than 25 MND clinical studies and the Tofersen study is the first study in which patients reported an improvement in their motor function.

The Argus: Tofersen helped people regain some motor function at 12 months (PA)Tofersen helped people regain some motor function after 12 months (PA)

“Never before have I heard patients say, ‘I’m doing things today that I couldn’t do a few months ago – walk around the house without a cane, climb the garden steps, write Christmas cards.’

“For me, this is an important treatment milestone.”

Around 5,000 people in the UK have MNDwith 2% developing the condition due to a defective SOD1 gene.

Chris McDermott, professor of translational neurology at SITraN and co-author of the study, said: “Although Tofersen is a treatment for only 2% of people living with Tofersen MNDwe learned a lot from conducting this clinical trial that will help us conduct smarter and faster clinical trials in the future.”

Two-thirds of the participants were randomly assigned to receive eight doses of Tofersen over a 24-week period.

They were assessed to assess motor function in swallowing and speaking, breathing, fine motor skills, and gross motor skills.

They also gave samples of spinal fluid so researchers could measure associated protein levels MND.

Results showed that the drug did not improve motor control and muscle strength at six months, but patients reported better patient mobility and lung function at 12 months.

Improvements were seen in the MND Biomarkers in the patients’ cerebrospinal fluid after six months.

What is motor neuron disease?

On the NHS website It says: “Motor neuron disease (MND) is a rare disorder that affects the brain and nerves. It causes a weakness that gets worse over time.”

It can affect a person’s ability to walk, talk, use their arms and hands, or breathe. Scientists hope a new drug could help slow motor neuron disease

Fry Electronics Team

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