Hundreds more children suffering from spinal muscular atrophy (SMA) in England are set to gain access to life-changing treatments following their approval for routine use on the NHS. The National Institute for Health and Care Excellence (NICE) has endorsed the drugs nusinersen (Spinraza) and risdiplam (Evrysdi), concluding that they offer substantial benefits by improving survival rates, slowing disease progression, and helping individuals maintain their independence.
Previous Access and Impact
These medications were previously available through a special access programme. NHS England reports that they have already helped 73 children with severe SMA (Type 1) survive to age five or older. Among them is Ezra Thorman, whose mother stated the treatment fundamentally changed the course of his life.
Understanding SMA
SMA is a genetic condition causing severe muscle weakness that can affect breathing, swallowing, and movement. Without treatment, babies suffering from the most severe form are often not expected to live beyond two years.
Newborn Screening Study
The approval coincides with a new study set to screen approximately 750,000 newborns in England for SMA, assessing the feasibility of adding it to post-birth baby checks. This measure was campaigned for by pop star Jesy Nelson after her twins were diagnosed with the condition.
