14 January 2026 – Update from the Tofersen Summit, Parliament

SOD1 MND: National Failure, National Solution Needed – Now.

14.1.26 the NICE website was updated to state that, following discussions with the drug company Biogen, the evaluation of Tofersen is now due to start in late March 2026, with submissions expected in early June 2026.
For people with SOD1 Motor Neuron Disease, that timeline is not neutral.
It is devastating.
The reality
SOD1 MND is one of the rarest, most aggressive, and most precisely understood genetic forms of MND.
It affects children, teenagers, and young adults, as well as older adults.
A treatment exists.
Tofersen can slow progression, stabilise function, and in some cases partially reverse damage.
It is:
Licensed
Used safely worldwide for over five years
Provided free to the NHS under the Biogen scheme
In the UK, access depends on postcode and the capacity of treating units.
That capacity has not been properly planned or funded.
Delivery has relied heavily on clinician goodwill, and that goodwill has now run out.
Currently, six centres deliver Tofersen.
Two further centres are hoping to start, but without national coordination, funding, and political support, hope does not translate into access. (25 MND centres in the UK) 
Around 18 eligible patients — including my 19-year-old step-daughter, Lillia, in Southampton — cannot access treatment not because it does not exist, but because there is no national pathway, co-ordination, or funded capacity. Approx 45 patients are receiving treatment.

This is not only a regional problem
It is a national failure.

❗ Where we are today
The drug is licensed
The drug is free
The expertise exists
Six centres are treating patients
Two more centres hope to start
Around 18 people are still being denied access
All are deteriorating weekly, with irreversible loss of function
There is:
No national register
No national co-ordination
No funded pathway to match patients to centres already offering treatment
Every week matters.
Every delay causes permanent harm.
🔍 How did this happen?
Tofersen was placed on the wrong NICE appraisal pathway nearly three years ago.
It was moved to the correct route — Highly Specialised Technologies (HST) — in October 2024.
The initial incorrect appraisal route has lost 4 years , leaving clinicians, providers, and families in limbo while patients continue to deteriorate. 

This situation is clinically indefensible.
It is ethically indefensible.

📣 What we are calling for — now

We call on Senior Politicians and Wes Streeting, the Department of Health, NHS England, and NICE to act immediately:

Provide emergency interim funding now
So existing centres can expand capacity and the two additional centres can start immediately, without relying on unpaid clinician goodwill.
Increase capacity in treating centres so that all patients receive fair and equitable access
Through proper funding, staffing, and senior political leadership to remove postcode and capacity-based inequality.

Establish a National SOD1 Co-ordination System (clinician-led)
To match every diagnosed patient to the most appropriate available centre.
Create a National SOD1 Register
So no patient is ever missed, unidentified, or left behind again.
Guarantee treatment for all eligible patients without further delay, as close to home as possible
Minimising travel burden, reducing inequality, and preventing avoidable physical and emotional harm.

💔 This must not drift
These people are not expendable. 

The treatment exists.
The expertise exists.
The drug is free.
What is missing is capacity, co-ordination, and leadership.

✍️ Please sign and share this petition

We need urgent action to:
End the postcode and capacity lottery
Protect the 18+ people currently denied treatment
Ensure every person with SOD1 MND has immediate access to care
Lives. Mobility. Dignity. Time.
These are not abstract outcomes.
They are being lost every week.
Thank you.

Dr Rachel Jakeman
MBBS MRCGP DFFP DRCOG
GP and step-mum to Lillia
Mum to Edie, Will and Harri