Routine NHS access to spinal muscular atrophy treatments offers new hope for patients and families
People living with spinal muscular atrophy (SMA) in England are set to gain routine access to two potentially life-changing treatments, following our publication of final draft guidance today.
From today everyone with this devastating condition now has a treatment option that is routinely available.
The final draft guidance recommends the use of nusinersen and risdiplam for certain people with SMA under the NHS. The decision marks a significant milestone after both medicines were previously made available through managed access agreements while additional evidence on their effectiveness was gathered.
SMA is a rare, inherited condition that causes progressive muscle weakness, affecting movement, breathing and swallowing. Without treatment, the condition can lead to severe disability and, in its most serious forms, early death—often before the age of two. Around 1,150 people in England are estimated to be living with SMA types 1 to 3.
Our recommendation reflects strong evidence that both treatments can improve survival rates, slow the time it takes for the disease to get worse and help patients maintain independence and quality of life. Clinical findings also show the therapies can reduce the need for permanent ventilation and support children in reaching key developmental milestones such as sitting, standing and walking.
We’ve recommended nusinersen 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.
Helen Knight, NICE’s director of medicines evaluation, said: “Spinal muscular atrophy is a devastating condition that affects every aspect of daily life for people living with it and for their families. After carefully reviewing the latest evidence and real-world experience from the NHS, our independent committee concluded that nusinersen and risdiplam can offer substantial, life‑changing benefits for many people with SMA.
“These treatments can help people live longer, maintain their independence, communication and participation in everyday life and reduce the need for hospital care.
By recommending routine NHS access to nusinersen and risdiplam, NICE has recognised both the severity of SMA and the transformational impact these treatments can have on people’s lives. For patients and families affected by SMA, the guidance represents a major step forward—offering greater certainty, improved quality of life and renewed hope for the future.
Public Health Minister, Sharon Hodgson said: “Spinal muscular atrophy can have a profound impact on every aspect of a person’s life, particularly for children and young people.
“Today’s final draft guidance is welcome news for families across the country and represents an important milestone in improving access to innovative NHS treatments for rare diseases.
“Drawing on both clinical evidence and the real-world experiences of patients already receiving treatment, this decision will help ensure more people with SMA can access the care and support they need to live fuller, more independent lives.”
“These treatments can help people live longer, preserve abilities that support independence and participation in everyday life, and reduce the need for hospital care.”
Data collected during the managed access period highlighted a range of additional benefits from the treatments. Patients reported improved physical and mental wellbeing, fewer emergency hospital admissions—particularly for respiratory infections—and greater participation in education, employment and social activities.
The impact has also been felt by carers, who reported better mental health, improved sleep and an increased ability to balance caring responsibilities with work and family life.
The independent committee also noted that even small improvements—such as maintaining movement in the hands or arms—can significantly improve independence, allowing patients to use technology, communicate and engage more easily in daily activities. Stabilising the condition, rather than reversing it, was also recognised as a meaningful and valuable outcome in a progressive disease like SMA.
There is no single treatment approach suitable for all SMA patients. Our final draft guidance supports access to both therapies to allow clinicians, patients and families to choose the option best suited to individual needs.