Hundreds more children with spinal muscular atrophy (SMA) will now have routine, long-term access to two transformative therapies on the NHS in England. From today, nusinersen and risdiplam will be routinely available after an earlier access programme that collected real‑world evidence, giving thousands of families greater certainty about ongoing care.
NHS England says it has secured a commercial agreement to support lifelong treatment for eligible patients. Data from the SMA REACH UK national registry show that these therapies have already substantially improved outcomes: 73 children with severe Type 1 SMA are now known to have survived to age five or older — more than half of those treated — whereas untreated Type 1 is typically fatal before age two.
Nusinersen (Spinraza®, Biogen) is delivered by injection into the spinal fluid at regular intervals. Risdiplam (Evrysdi®, Roche) is an oral medicine available as a daily syrup or tablet that can be taken at home. The NHS introduced nusinersen under an early access scheme in May 2019 and risdiplam under a similar time-limited arrangement in 2021 while longer-term evidence was gathered.
Real-world results collected over seven years show that access to disease-modifying treatment has changed the SMA journey for many children and families. Treated children are living longer and achieving milestones that were once unlikely, such as sitting, standing, walking and starting mainstream school.
The story of Ezra Thorman illustrates that change. Diagnosed with Type 1 SMA as an infant, Ezra began nusinersen at five months through an early access programme. Now nine years old and in Year 4, his mother says early treatment fundamentally changed his life and made it possible for him to attend mainstream school and play with peers.
SMA is a rare genetic condition that causes progressive muscle weakness and loss of movement and can affect breathing and swallowing. It is commonly classified by age and severity: Type 1 presents before six months, Type 2 between six and 17 months, Type 3 between 18 months and 17 years, and Type 4 in adults. About 70 babies are born with SMA in the UK each year, and there are an estimated 1,150 people living with Types 1–3 in England.
Today’s decision means all three approved disease-modifying options are now routinely commissioned by the NHS in England: nusinersen, risdiplam and the one‑off gene therapy on the market, Zolgensma®. SMA REACH UK data also report more than 350 people receiving treatment who are aged five or older: 107 treated with nusinersen, 200 with risdiplam and 45 with Zolgensma.
NHS clinical leaders highlighted the profound impact of these treatments. They note that families who once faced the prospect of losing a child before their second birthday now have hope that their children will reach childhood milestones and live longer, fuller lives. Patient groups and clinicians who campaigned for long‑term access said the decision reflects the strength of lived experience and real‑world evidence in shaping care.
As part of the agreement negotiated by NHS England, a recently approved higher‑dose nusinersen regimen will also be provided. The new dosing reduces the number of initial injections required — halving starting doses from four to two — before moving to a maintenance injection every four months.
Manufacturers and patient organisations welcomed the move. They said routine commissioning provides stability for families and recognises the benefits seen outside clinical trials, while also enabling continued monitoring of outcomes to improve care further.
NHS England stressed that although this is a major step forward, more work remains to improve outcomes for people with SMA across all ages. Continued data collection, early diagnosis and timely access to therapy will remain priorities to ensure the best possible quality of life for children and adults living with SMA.