Mum with rare MND fights for access to life-extending drug

A mother from West Lothian living with a rare form of Motor Neurone Disease is calling for access to a treatment she says could prolong – or even save – her life.

Angela Brown, from Bathgate, is among the small number of people diagnosed with MND who carry the SOD1 gene variation.

Around 2% of patients have this form of the disease, which progresses rapidly and currently has no widely available treatment in Scotland.

Just over a year on from her diagnosis, Angela says the condition is already changing her day-to-day life. Once passionate about creating bouquets for her business, she is now finding that work increasingly difficult.

Her diagnosis also carries painful memories. Six years ago, her brother Tam died from the same gene variation at the age of 45.

Now 47, Angela says she had long suspected something was wrong before being diagnosed.

She told STV News: “Watching somebody decline is heartbreaking, totally broke my mum and dad’s hearts, which is why it was difficult sharing the news with them, because it’s my mum’s side of the family that carries the gene.

“She almost feels as though it’s her fault and thinks ‘how can this happen again to one of us’.”

There is now hope in the form of a drug called Tofersen – the first treatment in more than 30 years proven to slow the progression of SOD1-related MND. However, while it is available free of charge to patients in England and Wales, it is not currently accessible in Scotland.

Angela says that reality is difficult to accept, particularly given her family’s history.

She said: “My brother worked so hard, saying ‘I know it’s never going to help me but it can help other people’.

“I say he started the fight and I want to finish it. My kids could have it (the gene), it might not help me in my lifetime, I might need to do drastic things like move to England to be able to get treatment, and that’s the reality.”

Campaigners are now urging the Scottish Government to take action to make the drug available.

Nicola Roseman, from the My Name’5 Doddie Foundation, said: “To have that hope taken away is just unacceptable.

“We have to look at how we do this quicker. We know that there’s the skillset out there. It’s not an easy treatment to give – you would require a lumber puncture once a month, so that does take time and it takes resources.

“We are ready to provide resources and funding, financial aid, to enable this to happen.”

The Scottish Medicines Consortium says it has not yet received a submission for the drug from pharmaceutical company Biogen, which plans to submit tofersen for approval in the second half of this year.

A spokesperson for the Scottish Medicines Consortium said: “The remit of the Scottish Medicines Consortium (SMC) is to provide advice to NHS Boards across Scotland about the clinical and cost-effectiveness of new medicines.

“SMC is not involved in early access schemes so is unable to comment on these.”

Meanwhile, a spokesperson for Biogen said: “Biogen recognises the significant unmet need for people living with SOD-1 ALS and the progressive, devastating nature of the disease.

“Since 2021 Biogen has made tofersen available free of charge to eligible patients in Scotland, and across the United Kingdom, through an early access programme, ensuring patients can benefit from treatment while formal NHS funding decisions are ongoing.

“Under this programme, Biogen supplies the medicine free of charge, while the NHS covers the administration and clinical resources needed to deliver the treatment.

“Biogen plans to submit tofersen to the Scottish Medicines Consortium (SMC) in the second half of 2026. While our application under the SMC’s ultra-orphan route was not accepted last year, we remain fully committed to progressing access for people living with SOD-1 ALS and will move forward under the SMC’s orphan pathway.

“Tofersen is now nationally reimbursed for eligible patients in ten countries including Germany, Spain, The Netherlands, Poland, Slovenia, Luxembourg and Lithuania.”

A Scottish Government spokesperson said: “As previously stated, the Scottish Government is committed to ensuring that people living with conditions like Motor Neurone Disease (MND) have access to high-quality care and support, and benefit from healthcare services that are safe, effective and person-centred.

“We have a clear and consistent route in Scotland for licensed medicines to be appraised through the Scottish Medicines Consortium (SMC). The SMC has not yet received a submission for Tofersen and would welcome a submission from the pharmaceutical company at any time.

“A pharmaceutical company may choose to offer a medicine free of charge prior to appraisal, sometimes known as an Early Access Programme (EAP). While such schemes provide the medicine at no cost, they do not normally cover the additional resource needed to support service implementation.”

Angela’s niece and nephew are fundraising for The My Name’5 Doddie Foundation and you can donate here.