People living with spinal muscular atrophy in England are set to gain routine access to two treatments after the National Institute for Health and Care Excellence published final draft guidance recommending nusinersen and risdiplam for certain patients under the NHS. The decision, announced on 14 May 2026, marks a significant shift for families affected by the rare inherited condition, which causes progressive muscle weakness and can affect movement, breathing and swallowing.
Routine access follows managed use and additional evidence gathering
NICE said both medicines had previously been available through managed access agreements while more evidence on their effectiveness was collected. With the new guidance, the treatments are now recommended for routine NHS use in England for certain people with SMA, offering a treatment option that the organisation described as potentially life-changing.
The announcement is especially significant because SMA can lead to severe disability and, in its most serious forms, early death, often before the age of two. NICE said the guidance reflects the seriousness of the condition and the impact these therapies may have on patients’ lives and on the support needs of their families.
A milestone for rare disease care in the NHS
The decision adds to a recent run of NICE recommendations for rare conditions, but this update stands out because it opens the door to routine access for two established treatments rather than a single new therapy. According to NICE, the move should help ensure more people with SMA can access care and support that may help them live fuller and more independent lives.
For patients and clinicians, the practical importance is straightforward: a disease that has long demanded specialised, ongoing management now has treatment options that can be provided more routinely within the NHS. For families, the news represents another step toward reducing uncertainty in the care pathway for a devastating neurological condition.
As the guidance is implemented, attention will turn to how quickly eligible patients can be identified and started on treatment across England. For now, the recommendation signals a notable milestone in the NHS response to one of the most serious rare diseases affecting children and adults.
Source: NICE news release
