NICE has recommended routine NHS access in England to two treatments for spinal muscular atrophy, in a move the organisation says could change the outlook for many patients and families living with the rare condition.
Two medicines move from managed access to routine use
Final draft guidance published on 14 May 2026 recommends nusinersen and risdiplam for certain people with spinal muscular atrophy, or SMA. The decision marks a shift from managed access agreements to routine availability under the NHS, after additional evidence on effectiveness was gathered.
NICE said the treatments are now available from today for people who meet the guidance criteria. The agency described the decision as a significant milestone for a condition that can cause progressive muscle weakness and, in its most serious forms, early death. NICE announcement
What the guidance says about SMA
SMA is a rare, inherited condition that affects movement, breathing and swallowing. NICE said that without treatment it can lead to severe disability and, in some cases, death before the age of two. Around 1,150 people in England are estimated to be living with SMA types 1 to 3.
The guidance says nusinersen can be used for both pre-symptomatic and symptomatic SMA in 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.
Evidence points to longer survival and better quality of life
NICE said the recommendation reflects evidence that both treatments can improve survival rates, slow disease progression and help patients maintain independence and quality of life. The agency also said clinical findings show the therapies can reduce the need for permanent ventilation and help children reach developmental milestones such as sitting, standing and walking.
During the managed access period, data collected showed additional benefits, including improved physical and mental wellbeing, fewer emergency hospital admissions, and greater participation in education, employment and social activities. Carers also reported better mental health, improved sleep and a greater ability to balance caring with work and family life.
Helen Knight, NICE’s director of medicines evaluation, said the independent committee concluded that nusinersen and risdiplam can offer substantial, life-changing benefits for many people with SMA. Public Health Minister Sharon Hodgson said the decision is welcome news for families and an important milestone in improving access to innovative NHS treatments for rare diseases.
A wider choice for clinicians and families
NICE said there is no single treatment approach suitable for all SMA patients, and that its final draft guidance supports access to both therapies so clinicians, patients and families can choose the option best suited to individual needs. The organisation said even small improvements, such as maintaining movement in the hands or arms, can make a meaningful difference in daily life.
The guidance adds to a growing set of NHS decisions aimed at improving access to medicines for rare diseases, while also reflecting the value of evidence gathered from real-world use. For patients and families affected by SMA, NICE said the recommendation represents a major step forward and offers renewed hope for the future.
