Hundreds more children with spinal muscular atrophy (SMA) can now access life-changing NHS treatments after the National Institute for Health and Care Excellence (Nice) approved two drugs for routine use across England. The decision ends years of uncertainty for families who had relied on special access programmes while evidence was gathered, and marks a significant expansion of care for the rare genetic condition.
Nice’s final draft guidance endorses both nusinersen (Spinraza, made by Biogen) and risdiplam (Evrysdi, made by Roche) for widespread NHS use. The watchdog said the drugs can improve survival rates, slow disease progression and help people maintain their independence. NHS England has also secured a commercial agreement with the manufacturers to guarantee lifelong treatment for eligible patients.
Understanding SMA and the new therapies
Spinal muscular atrophy is a genetic disorder that causes severe, progressive muscle weakness. It affects movement, breathing and swallowing. Without treatment, babies born with the most severe form — Type 1 — are unlikely to live beyond two years. Around 70 babies are born with SMA each year in the UK, and approximately 1,150 people live with the condition in England.
Nusinersen was the first disease-modifying therapy for SMA. It is given via a periodic spinal injection. A new formulation, recently approved, reduces the number of initial doses from four to two before moving to a maintenance injection every four months. Risdiplam is taken daily by mouth as a syrup or pill and can be administered at home. Both drugs work by increasing production of the survival motor neuron (SMN) protein, which is deficient in people with SMA, thereby slowing or stopping the muscle damage that defines the disease.
A third therapy, the one-off gene therapy Zolgensma, is already routinely commissioned by the NHS in England for children with Type 1 SMA, including those diagnosed before symptoms appear. Zolgensma, which costs £1.79 million per dose at list price (a discounted rate was agreed with the NHS), delivers a working copy of the missing SMN1 gene.
Indirect comparisons of real-world data suggest risdiplam may offer better long-term outcomes than nusinersen in Type 1 SMA. Studies indicate that risdiplam-treated children had a 78% lower death rate, an 81% lower rate of permanent ventilation and a 57% lower rate of serious adverse events compared with those on nusinersen. Risdiplam also appears to produce sustained improvements in motor function, while nusinersen may plateau after around 26 months. However, experts caution that head-to-head trials are still needed. Real-world evidence also shows that switching from nusinersen to risdiplam is safe and can lead to maintained or improved motor outcomes, particularly in upper limb function.
Real-world impact on children and families
Landmark data from the SMA REACH UK study — a national database — show that these therapies have helped 73 children with severe Type 1 SMA survive to age five or older. Overall, more than 350 people with SMA receiving treatment in England are now aged five and over: 107 have been treated with nusinersen, 200 with risdiplam and 45 with Zolgensma.
The difference treatment can make is illustrated by Ezra Thorman, now nine, from Ramsgate, Kent. Diagnosed with SMA Type 1 in 2016, he received nusinersen at five months old through an early NHS access programme. His mother, Portia Thorman, said: “His SMA Type 1 diagnosis in 2016 came with a life expectancy of under two years old. The hope for a future this bright felt out of reach, however accessing treatment at just five months old fundamentally changed the course of his life.” Ezra now attends mainstream school in Year 4.
Professor James Palmer, national medical director for specialised services at NHS England, described the treatments as “a phenomenal step forward in care”. He added: “For parents who faced the unimaginable pain of thinking their child would not reach their second birthday, they now have hope of seeing them walk to school and play with their friends.”
Helen Knight, Nice’s director of medicines evaluation, said the independent committee concluded that the drugs “can offer substantial, life-changing benefits for many people with SMA”, helping them live longer, maintain independence and reduce the need for hospital care. Public health minister Sharon Hodgson said the decision, drawing on clinical evidence and real-world patient experiences, “will help ensure more people with SMA can access the care and support they need to live fuller, more independent lives”. Giles Lomax, chief executive of SMA UK, called it “a historic moment for the SMA community” after years of determined campaigning.
Screening study aims to catch SMA at birth
The announcement comes alongside a major new study that could transform how SMA is detected. Around 750,000 newborns in England will be checked for the condition as part of a research project to assess whether SMA should be added to the routine heel-prick blood test given to all babies shortly after birth.
The study, funded by the National Institute for Health and Care Research (NIHR) and led by scientists at the University of Oxford under Professor Laurent Servais, will evaluate the feasibility, acceptability, effectiveness and cost-effectiveness of adding SMA to the newborn blood spot screening. It will begin in seven NHS newborn screening laboratories in August 2026, with possible nationwide extension. Final results are expected in 2031.
Scotland has already become the first part of the UK to routinely screen newborns for SMA, launching a two-year pilot in March 2026 funded by the Scottish Government and Novartis. The UK has been noted as lagging behind many European countries that already offer SMA screening.
Pop star Jesy Nelson, whose twins Ocean Jade and Story Monroe Nelson were diagnosed with the condition, has campaigned publicly for screening to be rolled out. Early diagnosis allows treatment to begin before symptoms appear — considered far more effective than treating after muscle damage has already occurred. Modelling suggests that adding SMA screening to the existing heel-prick test could lead to better outcomes and lower overall costs, with an estimated cost of £4 to £5 per baby. A large UK survey has shown widespread support among families, healthcare professionals and the public for the move.