Sign our petition to help people living with SOD1 MND access a life-changing treatment
For the 2% of people with MND whose disease is caused by a fault in the SOD1 gene, there is a ray of hope. A groundbreaking new treatment, tofersen, has been developed to treat this type of MND. It can significantly slow down the progression of MND and in some cases stop it altogether – something we have never seen before in an MND treatment.
For people like Seckin McGuirk, who is living with SOD1 MND, tofersen offers new hope of slower disease progression, longer life expectancy, and more time to enjoy life with her family and friends.
But she’s been told she can’t have tofersen, due to NHS resource constraints.
Tofersen is currently being assessed to see if it will be paid for by the NHS, but that will take months. In the meantime, tofersen has been made available to patients free of charge through an Early Access Programme (EAP).
This has led to some of the people who need it getting access, and many of these people have seen their condition stabilise. But some people who desperately need this treatment are missing out, because their local services do not have capacity to give it to them via the required monthly lumbar puncture – even though the drug itself is available for free.
Sadly, Seckin was given the terrible news that she cannot currently be given tofersen. She and her family have to cope with knowing that there is a freely available drug that could significantly extend her life – but she cannot have it.
That’s why we are calling on the Government to take urgent action so that everyone who needs tofersen can access the EAP. It’s not right that some people who desperately need this drug can’t receive it. People with MND don’t have time to wait. A solution is desperately needed to break down the barriers to access – and it is needed now.
Please sign our petition addressed to Karin Smyth MP, Minister of State at the Department of Health and Social Care, urging her to intervene immediately so nobody misses out on this vital treatment.
What is tofersen?
Tofersen is a groundbreaking new treatment that is effective for the 2% of people with MND whose disease is caused by a fault in the SOD1 gene.
Is tofersen available on the NHS?
Tofersen is currently (as of May 2025) in the process of being licensed and then assessed to become available on the NHS. This process will take several months at least. In the meantime, the drug is being offered to the NHS at zero cost.
What was the tofersen campaign win last year?
In October 2024 we launched a petition asking NICE to reconsider how they categorise tofersen, to give it the best chance of passing assessment and being made available on the NHS.
On 18 November 2024 NICE announced that – as our petition had been asking – they had reconsidered their decision, and agreed to assess tofersen via the Highly Specialised Technology (HST) route.
This means that there’s a much greater chance of tofersen becoming available on the NHS.
More than 15,000 people signed our petition in just a few weeks, sending NICE a powerful message about how important tofersen is – a groundbreaking new treatment for the 2% of people with MND who have an alteration in the SOD1 gene. And NICE listened.
Who can access tofersen currently?
Tofersen is currently being assessed to become available on the NHS, but that will take several months at least. In the meantime, the manufacturer has made tofersen available to patients free of charge through an Early Access Programme (EAP), as long as the NHS provides the staff time and resources.
But some people who desperately need this treatment are missing out, because their local services don’t have capacity to give it to them via the required monthly lumbar puncture – even though the drug itself is available for free.
