Beyond Mobility: Why Holding the Line is a Major Win in SMA Treatment
When the NICE committee reviews access to risdiplam and Spinraza for Spinal Muscular Atrophy (SMA), SMA UK along with our partners Treat SMA and MDUK are urging them to look past just big improvements. For a condition that always gets worse, like SMA, stabilisation, or simply stopping the disease from progressing, is a hugely effective outcome.
All SMA Patient Groups are together in arguing that the true value of these treatments goes Beyond Mobility.
The Hidden Value of Hitting the Pause Button
If you look at health assessments, there’s a pressure to see massive gains. But SMA is a progressive disease, meaning it relentlessly destroys motor neurons and weakens muscles. If you don’t intervene, decline is certain.
- A Win for Health: Every motor neuron saved is a lifetime of function preserved. Stabilising function means maintaining the ability to breathe, swallow safely, or sit up independently. This isn’t just “staying the same”—it’s retaining vital quality of life and preventing the severe complications that lead to aggressive, painful, and often continuous care.
- A Smart Economic Choice: Not treating SMA is incredibly expensive in the long run. Decline means more hospital visits, more complex equipment, and a higher burden on the health and social care systems. By stabilising the condition and preventing that decline, these therapies are reducing long-term care costs.
We urge NICE to understand that in the fight against a debilitating, progressive disease, stabilising the patient is a profound and effective medical victory.
Technology Appraisal Committee Meeting
The registration facility for the committee meeting for the above appraisal, to be held on Tuesday 11 November 2025, is now open on the NICE website. The meeting will be held virtually, using Zoom, a secure web conferencing tool. The decision on access to both treatments will be made after the 11th November. We will update as and when we are able.
You can find the links to register directly on Zoom on this page.
Registration will close at 4.00pm on Tuesday 4 November 2025.
As a public observer, you will be able to listen to the business of the meeting, except where confidential information is being discussed, however, you will not be able to:
- participate in committee discussions
- ask questions, take part in voting or put your views to members of the committee
For further information about meetings in public please refer to the following document: Common questions and answers about virtual committee meetings.
