NHS Approves Life-Saving Drugs for Children With Spinal Muscular Atrophy

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The National Institute for Health and Care Excellence (NICE) has approved two groundbreaking treatments for spinal muscular atrophy (SMA), a rare and often fatal genetic condition that causes progressive muscle weakness. The decision means hundreds of children across England, Wales, and Northern Ireland will now have routine access to nusinersen (Spinraza) and risdiplam (Evrysdi) through the NHS, marking a major shift from previous time-limited schemes.

SMA is typically diagnosed in infancy and, without treatment, can be fatal before age two. The most severe form, Type 1 SMA, affects motor neurons in the spinal cord, leading to rapid muscle degeneration, difficulty breathing, and swallowing challenges. The new NICE guidance ensures long-term availability of both therapies, offering families certainty and hope after years of uncertainty.

Life-Changing Impact for Children with SMA

NICE’s final draft guidance recommends that any patient who would benefit from either treatment can now receive it without restriction. This follows years of evidence collection through NHS access schemes, during which nusinersen was first offered in May 2019 and risdiplam in 2021. The move is based on emerging data from the SMA REACH UK study, which found that 73 children with Type 1 SMA—more than half of those treated—have now survived to age five or older, a milestone previously considered unlikely.

For parents of children with SMA, the approval represents a “lifeline.” Portia Thorman, whose nine-year-old son Ezra began nusinersen treatment at five months old, described the therapy as having “fundamentally changed the course of his life.” Ezra, now in Year 4 at a mainstream school in Kent, is one of the children who would have faced severe disability or early death without intervention.

How the Treatments Work

The two approved drugs target SMA differently. Nusinersen, developed by Biogen, is administered as a regular injection into the spinal fluid, while risdiplam, from Roche, is taken orally as a daily syrup or tablet. Both therapies modify the disease’s progression by addressing the underlying genetic defect, though they require lifelong treatment.

NHS England’s national medical director for specialised services, Prof James Palmer, emphasized the transformative potential of the treatments: “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.”

Broader Implications for Rare Disease Treatment

The approval reflects a broader trend in NHS policy toward rare diseases, where early access schemes have allowed real-world evidence to inform long-term funding decisions. Previously, both nusinersen and risdiplam were available only under time-limited arrangements while additional data was collected. The new guidance ensures continuous access, aligning with international standards for treating SMA.

While the decision is a major victory for patient advocacy groups, some experts note that challenges remain. Early diagnosis and equitable access across all regions will be critical to maximizing the therapies’ benefits. The NHS will also need to monitor long-term outcomes to refine guidelines further.

What Comes Next?

From today, eligible patients in England, Wales, and Northern Ireland can begin routine treatment with either nusinersen or risdiplam. The NHS has secured a commercial agreement to guarantee lifelong access, eliminating the previous uncertainty that families faced. Advocates hope this decision will accelerate research into other rare diseases and encourage earlier interventions.

For families affected by SMA, the approval offers not just medical relief but emotional reassurance. As one parent put it in recent statements, “This is more than a treatment—it’s a second chance at a future we were told we’d never have.”