The National Institute for Health and Care Excellence has approved two drugs to treat spinal muscular atrophy on the NHS, making them routinely available to all eligible patients in England, Wales and Northern Ireland from this week. The decision ends years of uncertainty over long-term access to treatments that clinicians say have fundamentally altered outcomes for children born with the condition.

Spinal muscular atrophy is a progressive genetic disorder that weakens the muscles and impairs the ability to move, breathe and swallow. In its most severe form, type 1 SMA, children historically died before their second birthday. The condition affects an estimated 1,150 under-18s in England and between 1,500 and 2,500 people of all ages across the UK. Around 70 babies are born with it each year.

The two drugs now cleared for routine use are nusinersen, sold under the brand name Spinraza, and risdiplam, sold as Evrysdi. Nusinersen is administered by injection into the spinal fluid at regular intervals and requires clinical supervision. Risdiplam is taken daily at home as a syrup or tablet, making it considerably more accessible for families managing the condition outside hospital settings. Both have been available on a limited basis since 2019 and 2021 respectively, but their long-term NHS availability was not guaranteed until now. NHS England has reached pricing agreements with manufacturers Biogen and Roche Pharmaceuticals to secure their routine supply.

The practical consequences of the drugs are documented in individual cases as much as in clinical data. Portia Thorman's son Ezra, now nine, was diagnosed with type 1 SMA in 2016 and began receiving nusinersen at five months old. Without it, his mother says, he would not have survived infancy. "He's alive. He would have been dead by the age of two without it," she said. Ezra spent the first years of his life cycling in and out of intensive care with respiratory infections. He now attends mainstream primary school in Ramsgate, Kent. "Starting mainstream school is a milestone we were once told he would never reach," his mother said.

SMA has attracted greater public attention since the singer Jesy Nelson disclosed last year that her twin daughters, born in May 2025, have the condition. Nelson has since campaigned for SMA to be included in the routine heel-prick blood test given to newborns. Researchers at the University of Oxford are currently assessing the feasibility and cost of doing so. NHS England has indicated it plans to begin an in-service evaluation of newborn screening in October, which could form the basis of a wider programme if the evidence supports it.

The response from NHS England and the regulator was measured but clear in its assessment of the decision's significance. Professor James Palmer, NHS England's national medical director for specialised services, said the treatments had delivered a step forward in care for children and families affected by a condition that had previously offered little hope. Helen Knight, Nice's director of medicines evaluation, said independent experts had determined the drugs could offer substantial benefits, including longer survival, greater independence and reduced reliance on hospital care. Giles Lomax, chief executive of SMA UK, described it as a historic moment for the patient community, noting that families had faced prolonged uncertainty over whether the treatments would remain accessible.

The scale of the population affected remains relatively small by the standards of NHS decision-making, but the severity of the condition gives the approval considerable weight. For children with type 1 SMA in particular, the question has not been whether these treatments improve quality of life. It has been whether families could rely on them being available. That question now has a definitive answer.