New data from the SMA Research and Clinical Hub (SMA REACH UK) database has shown that babies that are born with spinal muscular atrophy (SMA) are surviving for longer than two years thanks to NHS treatment advancements.
Each year in the UK, around 70 babies are born with SMA, a rare genetic condition that causes muscle weakness, progressive loss of movement and paralysis.
The new data has shown that 73% of children in the UK with SMA type 1 (SMA1), the most common form of the disease, are older than two years.
Previous US studies of the disease have estimated that less than 8% of children born with SMA1 survived to the age of 20 months without permanent ventilatory support prior to the availability of treatments.
The data extracted from the SMA REACH UK database at the end of 2022 showed that 118 out of 161 patients with SMA1 were aged over two years old and living without permanent ventilatory support, with many more children in the database who were under two years old at the time now expected to have survived beyond that milestone.
Amanda Pritchard, NHS chief executive, said: “It’s fantastic to see that more and more children diagnosed with this debilitating condition are living longer and with greater independence.”
A significant reduction in deaths from SMA1 in England was also shown, with only 11 recorded across the UK between March 2018 and March 2023. This is compared to around 25 each year between 2008 and 2017.
Professor James Palmer, national medical director of specialised services at NHS England, said: “A trio of new treatments are improving outcomes for NHS patients by slowing the progression of SMA, enabling a longer and better quality of life with family and friends.”
The new findings follow the recent roll-out of three new SMA medicines provided on the NHS: injectable drug Spinraza (nurinersen), gene therapy Zolgensma and oral treatment risdiplam, as well as health services commercial deals to secure access for SMA patients across the UK.
Spinraza was the first treatment to target the underlying causes of SMA, made available on the NHS in 2019.
The NHS has ensured access to treatment for patients with types 1, 2 and 3 SMA and said it hopes that all three treatments will continue to deliver improved outcomes as more data is collected.