NHS drugs go-ahead offers lifeline to children with rare muscle-wasting disease
Hundreds of children with a rare muscle-wasting disease will be able to receive two drugs that can improve their survival in a move parents hailed as a “lifeline”.The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug.The move means that anyone in England, Wales or Northern Ireland with spinal muscular atrophy will from Thursday be able to get either nusinersen, also known as Spinraza, or risdiplam, also known as Evrysdi, from the NHS.SMA is a progressive genetic disorder that causes severe muscle weakness and can affect the ability to move, breathe and swallow.Without treatment, patients face devastating consequences including profound disability and reduced life expectancy.
Children with the most severe form of SMA – type 1 – usually die before they reach two.Prof James Palmer, NHS England’s national medical director for specialised services, said: “These lifeline treatments have offered a phenomenal step forward in care for children and families affected by such a debilitating condition and it is fantastic that they will now be available on the NHS in the long term.“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, thanks to these lifechanging new therapies.”There are thought to be about 1,150 under-18s with SMA in England and between 1,500 and 2,500 of all ages across the UK as a whole.An estimated 70 babies are born with SMA in the UK each year.
Portia Thorman said the life of her nine-year-old son, Ezra, had been transformed by receiving nusinersen from just five months old,She added: “What difference did it make? He’s alive,He would have been dead by the age of two without it,He was one of the first people in the UK to get the drug,That was huge.
It was literally a lifeline for him.“For example, in the first three years of his life we were in and out of intensive care every time he got a cold.But now we stay at home when he has a cold, because the drug stopped the progression of his disease.”Ezra is now at primary school in Ramsgate, Kent.“Starting mainstream school is a milestone we were once told Ezra would never reach,” his mother said.
“His SMA type 1 diagnosis in 2016 came with a life expectancy of under two years.Accessing treatment at just five months old fundamentally changed the course of his life.”SMA has gained a high profile after Jesy Nelson, the former Little Mix singer, disclosed that her twin daughters born in May 2025 have the condition.She is campaigning for all newborns to be screened for SMA as part of the routine heel-prick blood test.University of Oxford scientists are assessing the feasibility and costs of doing so.
The NHS in England plans to start an “in-service evaluation” of newborn screening in October, which may pave the way for it to become part of newborn care.Nusinersen is administered as a regular injection into the patient’s spinal fluid while risdiplam is taken daily in syrup or tablet form at home.The drugs have been available in a limited way since 2019 and 2021 respectively.NHS England has struck a price agreement with their manufacturers – Biogen (nusinersen) and Roche Pharmaceuticals (risdiplam) – to make them routinely available to anyone who may benefit.Helen Knight, Nice’s director of medicines evaluation, said independent experts had considered the evidence and decided the drugs could “offer substantial, life-changing benefits for many people with SMA”.
She added: “These treatments can help people live longer, maintain their independence, communication and participation in everyday life and reduce the need for hospital care”,Giles Lomax, the chief executive of SMA UK, said: “This is a historic moment for the SMA community,After years of uncertainty, people can finally feel confident that these treatments will remain available on the NHS,”
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