In a significant breakthrough for families affected by spinal muscular atrophy (SMA), the National Institute for Health and Care Excellence (NICE) has sanctioned the use of two critical drugs, nusinersen and risdiplam, for patients under the age of 18. This decision, welcomed by parents and health professionals alike, promises to enhance the quality of life and survival prospects for children grappling with this rare, life-threatening condition.
Lifesaving Treatments Available on the NHS
From Thursday, children in England, Wales, and Northern Ireland diagnosed with spinal muscular atrophy will have the opportunity to receive either nusinersen, known commercially as Spinraza, or risdiplam, branded as Evrysdi, through the National Health Service. This approval marks a pivotal moment for approximately 1,150 children under 18 in England alone, with many more affected across the UK.
Spinal muscular atrophy is a progressive genetic disorder characterised by severe muscle weakness, which can severely hinder movement, breathing, and swallowing. Tragically, the most severe form of SMA, known as type 1, typically leads to a life expectancy of less than two years without intervention.
Professor James Palmer, NHS England’s national medical director for specialised services, expressed optimism regarding the impact of these treatments. “These lifeline therapies represent a remarkable leap forward in care for children and families facing such a debilitating condition. It is heartening to know that parents, who once feared their child would not reach their second birthday, can now envision a future where their children thrive, walk to school, and engage with their peers,” he remarked.
A Personal Journey: Ezra Thorman’s Story
Among those set to benefit from this new availability is nine-year-old Ezra Thorman, who has been receiving nusinersen since he was just five months old. His mother, Portia, has spoken candidly about the transformative impact the drug has had on their lives. “What difference did it make? He’s alive. Without it, he would have likely passed away by age two. Ezra was one of the first in the UK to receive this treatment, and it has been nothing short of a lifeline,” she stated.
Prior to starting treatment, Ezra frequently required intensive care whenever he contracted a common cold. Now, thanks to the medication, he can manage these illnesses at home. “Starting mainstream school is a milestone we were once told Ezra would never reach. His SMA type 1 diagnosis came with a life expectancy of under two years. Accessing treatment so early has fundamentally changed his life,” Portia added.
The Call for Newborn Screening
SMA has gained increased visibility in recent years, notably after Jesy Nelson, former member of the group Little Mix, revealed that her twin daughters were born with the condition in May 2025. Nelson has since been advocating for routine screening for SMA in newborns as part of the standard heel-prick blood test, a move currently under consideration by researchers at the University of Oxford.
The NHS in England plans to initiate an “in-service evaluation” of newborn screening for SMA in October, potentially paving the way for its inclusion in standard newborn care.
Treatment Administration and Availability
Nusinersen is administered via regular injections into the spinal fluid, while risdiplam is available in a daily oral form, either as syrup or tablet, allowing for at-home treatment. Both drugs have been accessible on a limited basis since 2019 and 2021, respectively. In a notable development, NHS England has negotiated pricing agreements with Biogen (for nusinersen) and Roche Pharmaceuticals (for risdiplam), ensuring that these vital treatments can be provided to all who may benefit.
Helen Knight, NICE’s director of medicines evaluation, highlighted the significance of this decision, stating that independent experts concluded these treatments offer substantial, life-changing benefits. “They can help individuals live longer, maintain independence, and reduce the need for hospital care,” she affirmed.
Giles Lomax, chief executive of SMA UK, echoed this sentiment, declaring that this is a historic moment for the SMA community. “After years of uncertainty, families can finally feel assured that these treatments will be available on the NHS,” he said.
Why it Matters
The approval of nusinersen and risdiplam for children with spinal muscular atrophy represents a monumental step forward in the fight against this devastating disease. Not only does it provide hope to families who have faced unimaginable challenges, but it also underscores the importance of accessible healthcare for all. This decision paves the way for a future where children like Ezra can thrive, ensuring that they not only survive but flourish in their formative years, highlighting the critical role of innovative treatments in transforming lives.
