People living with spinal muscular atrophy in England are set to gain routine NHS access to two treatments after NICE published final draft guidance on 14 May 2026. The recommendation covers nusinersen and risdiplam for certain patients, marking a significant step for a rare condition that can cause progressive muscle weakness, breathing problems and swallowing difficulties.
What the new guidance means for patients
NICE said the decision follows a review of the latest evidence and real-world experience from the NHS. The committee concluded that both medicines can offer substantial benefits for many people with spinal muscular atrophy, including improved survival, slower disease progression and better quality of life.
According to the guidance, nusinersen is recommended for both pre-symptomatic and symptomatic patients who have not responded successfully to gene therapy with onasemnogene abeparvovec. Risdiplam will be offered to patients with SMA types 1, 2 or 3 who have not benefited from that gene therapy, as well as for pre-symptomatic cases where gene therapy is unsuitable.
Why the decision matters
Spinal muscular atrophy is an inherited condition that can lead to severe disability and, in its most serious forms, early death, often before the age of two. NICE said around 1,150 people in England are estimated to be living with SMA types 1 to 3. The guidance also notes that the treatments can reduce the need for permanent ventilation and help children reach developmental milestones such as sitting, standing and walking.
Helen Knight, NICE’s director of medicines evaluation, said the committee concluded that nusinersen and risdiplam can offer life-changing benefits, helping people live longer and maintain independence, communication and participation in everyday life. Public Health Minister Sharon Hodgson said the decision represents an important milestone in improving access to innovative NHS treatments for rare diseases.
Data collected during the managed access period also pointed to wider benefits for patients and carers, including improved physical and mental wellbeing, fewer emergency hospital admissions and better sleep for carers. NICE said even small improvements in movement can have meaningful effects on independence, while stabilising a progressive disease can be a valuable outcome in itself.
The guidance does not set out a single treatment path for every patient. Instead, NICE says access to both therapies will give clinicians, patients and families more choice in finding the option best suited to individual needs. NICE announcement
With routine NHS access now recommended, the decision could offer a more predictable route to treatment for families affected by a condition that requires lifelong management and, in many cases, early intervention.
