For parents raising a child with spinal muscular atrophy, the days can be measured in milestones that most families take for granted: a first step, a wave, a hug. SMA, a rare inherited condition that progressively destroys muscle strength, has a way of making even the smallest movements feel precious and uncertain.
That’s why the news coming out of NICE this week carries so much weight.
England’s health technology body has published final draft guidance recommending routine NHS access to two treatments, nusinersen and risdiplam, for people living with SMA. Around 1,150 people in England are estimated to be affected by SMA types 1 to 3, and from today, every one of them has a treatment option available on the NHS as standard.
From Trial Access to Routine Care
Both medicines have been available through managed access agreements for some time, meaning patients could access them while researchers continued collecting real-world evidence. That evidence-gathering period is now over, and the findings made the case compelling.
The data show that both treatments can improve survival rates, slow disease progression, and help patients hold onto independence and quality of life. The results are particularly striking: the therapies have supported kids in reaching developmental milestones like sitting, standing and walking achievements that, without treatment, may never have been possible.
NICE’s guidance is nuanced, recognising that no single treatment suits every patient.
Public Health Minister Sharon Hodgson echoed the significance of the moment, describing the guidance as “an important milestone in improving access to innovative NHS treatments for rare diseases.”
Nusinersen has been recommended for both pre-symptomatic and symptomatic SMA in patients who haven’t responded to gene therapy with onasemnogene abeparvovec.
Risdiplam will be offered to patients with SMA types 1, 2, or 3 who haven’t benefited from gene therapy, as well as to pre-symptomatic cases where gene therapy isn’t suitable.
Numbers and clinical outcomes only tell part of the story. What makes this guidance stand out is how seriously the independent NICE committee took the lived experience of patients and their families during the managed access period.
Patients reported fewer emergency hospital admissions, particularly for respiratory infections. They described better physical and mental well-being and increased participation in education, work and social life. For many, treatment meant re-engaging with the world in ways they’d thought were behind them.
Carers, too, noticed a shift. Many reported sleeping better, managing their mental health more effectively, and finding it easier to balance caring alongside work and family life. When a loved one stabilises, the whole household breathes a little easier.
Helen Knight, NICE’s director of medicines evaluation, put it plainly: “Spinal muscular atrophy is a devastating condition that affects every aspect of daily life for people living with it and for their families.”
Stabilising a progressive condition, even without reversing it, is an outcome worth fighting for. NICE has recognised that. Now the NHS has too.
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