‘Truly remarkable’ drug offers hope to motor neurone disease patients

Scientists say they have slowed down and even reversed some of the relentless and devastating decline caused by motor neurone disease.

 

The treatment has so far only worked in 2% of patients, but has been described as “truly remarkable” and a “real moment of hope” for the whole disease.

 

The MND Association said there was “mounting confidence” in the therapy.

 

MND, also known as amyotrophic-lateral sclerosis (ALS) is caused by the death of the nerves that carry messages from the brain to a sufferer’s muscles, affecting their ability to move, talk and breathe.

 

The disease dramatically shortens people’s lives and most will die within two years of being diagnosed.

 

A mutation in a specific part of a sufferer’s genetic code leads to the production of a toxic form of the protein SOD1, which kills motor neurones.  These mutations cause around 2% of MND cases, but one in five of those that run in families.

 

The new trial involving 108 patients, used an innovative type of medicine called gene silencing.  The drug tofersen effectively mutes the defective DNA so less SOD1 is produced.

 

Dr Brian Dickie, MND Association’s research director, said the treatment had the “potential to deliver a significant benefit” for a relatively rare group of people with the disease.  The big question, he added, was whether to give the drug in the earliest stages of the disease, when it “may be even more effective”, or even to healthy people with the SOD1 mutation to “prevent the onset of the disease”.

 

Chadwick Lawrence’s specialist team of clinical negligence lawyers have years of experience in medical negligence claims, providing support and guidance after life-changing events.  Not only do we represent clients in Yorkshire, but because of our reputation we also represent clients nationally.

 

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Posted by Karen Motley, Clinical Negligence Department, Chadwick Lawrence LLP (tonymay@chadlaw.co.uk ), medical negligence lawyers and clinical negligence solicitors in Huddersfield, Leeds, Wakefield and Halifax, West Yorkshire.

 

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