NICE has recommended routine NHS access to two spinal muscular atrophy treatments in England, marking what the organisation described as a significant step forward for people living with the rare inherited condition and their families. The final draft guidance covers nusinersen and risdiplam for certain patients, after both medicines were previously available through managed access agreements while more evidence was collected.
Two therapies, one goal: slowing a devastating disease
According to NICE, spinal muscular atrophy causes progressive muscle weakness that affects movement, breathing and swallowing, and in its most serious forms can lead to early death, often before the age of two. The organisation estimates that around 1,150 people in England are living with SMA types 1 to 3. Its latest recommendation says both treatments can improve survival rates, slow disease progression and help people maintain independence and quality of life. NICE also said the therapies may reduce the need for permanent ventilation and help children reach key developmental milestones such as sitting, standing and walking. NICE statement
How the guidance is expected to be used
The guidance recommends nusinersen for both pre-symptomatic and symptomatic people with SMA who have not responded successfully to gene therapy with onasemnogene abeparvovec. Risdiplam is recommended for people 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. NICE said there is no single treatment approach that fits every patient, and that the final draft guidance is intended to support access to both therapies so clinicians, patients and families can choose the option best suited to individual needs.
The decision was backed by data gathered during the managed access period, which NICE said highlighted additional benefits beyond the clinical outcomes. Patients reported better physical and mental wellbeing, fewer emergency hospital admissions, particularly for respiratory infections, and greater participation in education, employment and social activities. Carers also reported improved mental health, better sleep and a better balance between caring responsibilities and work or family life.
Families and clinicians see a major milestone
NICE director of medicines evaluation Helen Knight said the independent committee had concluded that nusinersen and risdiplam can offer substantial, life-changing benefits for many people with SMA. She added that the treatments can help people live longer, maintain their independence, communication and participation in everyday life, and reduce the need for hospital care. Public Health Minister Sharon Hodgson described the guidance as welcome news for families across the country and an important milestone in improving access to innovative NHS treatments for rare diseases. The recommendation now moves England closer to routine use of both medicines for eligible patients. Read the guidance summary
For many families affected by SMA, the latest decision offers a clearer treatment pathway and a chance to preserve more of the function and independence that the disease can take away. NICE said even small improvements, such as maintaining movement in the hands or arms, can make a meaningful difference in daily life, communication and the use of technology. The committee’s conclusion reflects a broader shift toward recognising stabilisation as a valuable outcome in a progressive condition where slowing decline can be life-changing.
