For the first time, children and adults with spinal muscular atrophy (SMA) will have access to two life-changing treatments on the NHS, following a landmark decision by the National Institute for Health and Care Excellence (NICE). The approval of risdiplam and onasemnogene abeparvovec marks a turning point for families affected by this devastating genetic condition, which has historically offered very limited treatment options.
Risdiplam (Evrysdi), taken as an oral medication, has been recommended for adults and children with SMA types 1, 2, and 3. This includes both those showing early symptoms and those who haven't yet developed signs of the condition, as well as patients who have previously received other SMA treatments. Meanwhile, onasemnogene abeparvovec (Zolgensma) – a one-off gene therapy – is now available for babies and young children with the most severe form, SMA type 1. These recommendations follow careful managed access agreements and commercial negotiations that have secured the treatments at a price the NHS considers cost-effective.
SMA is a rare, inherited condition that causes progressive muscle weakness and loss of movement due to the breakdown of motor neurones in the spinal cord. Around 1 in 10,000 babies born in the UK are affected, making it one of the leading genetic causes of infant death. The condition varies in severity, with type 1 being the most serious form, typically diagnosed in the first few months of life.
Until now, treatment options for SMA have been extremely limited, leaving many patients facing a future of increasing disability and shortened life expectancy. These new therapies represent a fundamental shift in how the condition can be managed. Clinical trials suggest they can improve motor function, reduce the need for breathing support, and significantly enhance quality of life for patients and their families. Having both an oral treatment and a gene therapy available gives doctors greater flexibility to tailor care to each patient's specific needs and stage of disease.
This NICE decision highlights the vital importance of research into rare diseases and demonstrates how collaboration between pharmaceutical companies and the NHS can make cutting-edge treatments accessible to those who need them most. For the estimated 2,500 people living with SMA in the UK, these recommendations offer genuine hope and the prospect of treatments that can meaningfully change the course of their condition. If you or a family member has SMA and would like to know more about these treatment options, speak to your GP or specialist team about whether they might be suitable for your individual circumstances.