NICE has recommended routine NHS access to nusinersen and risdiplam for certain people with spinal muscular atrophy (SMA) in England, marking a major shift in care for a rare inherited condition that can cause progressive muscle weakness and, in severe cases, early death.
Two treatments move from managed access to routine availability
The final draft guidance, published on 14 May 2026, means the two medicines are now routinely available for eligible patients after previously being offered through managed access agreements while additional evidence was collected. NICE said the decision reflects strong evidence that both treatments can improve survival, slow disease progression and help patients maintain independence and quality of life.
SMA affects movement, breathing and swallowing. NICE estimates around 1,150 people in England are living with SMA types 1 to 3, and without treatment the condition can lead to severe disability and, in its most serious forms, early death, often before the age of two.
What the guidance says for patients
NICE said nusinersen will be used to treat 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.
The agency also said clinical findings show the therapies can reduce the need for permanent ventilation and support children in reaching milestones such as sitting, standing and walking. Data gathered during the managed access period pointed to further benefits, including improved physical and mental wellbeing, fewer emergency hospital admissions, especially for respiratory infections, and greater participation in education, employment and social activities.
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.
NICE also noted that carers reported better mental health, improved sleep and a greater ability to balance caring responsibilities with work and family life. The committee said even small improvements, such as maintaining movement in the hands or arms, can significantly improve independence, and that stabilising a progressive disease can be a meaningful outcome.
There is no single treatment approach suitable for all people with SMA, and NICE said the guidance supports access to both therapies so clinicians, patients and families can choose the option best suited to individual needs. The recommendation adds to a series of recent NHS decisions aimed at improving access to treatments for severe and rare conditions across England.
Source: NICE
