The new data, from the national SMA Research and Clinical Hub (SMA REACH UK) database, also suggests a significant reduction in deaths from the most severe form of SMA, SMA1, with just 11 deaths in total recorded across the UK between March 2018-March 2023 – compared to around 25 deaths each year in England between 2008-2017, according to ONS data.
The data predominantly demonstrates the initial impact of injectable drug nurinersen (Spinraza®) – the first treatment to target the underlying cause of SMA – which was made available on the NHS in England from Summer 2019.
With the introduction of two other treatments – gene therapy Zolgensma® and oral treatment risdiplam secured in 2021 - the NHS has ensured access to treatment for patients with types 1, 2 and 3 SMA, and it is hoped all three treatments will continue to deliver improved outcomes as more data is collected. MDUK played a crucial role in securing access to these treatments, working in partnership with clinicians, families and other patient groups to advocate for NICE and the Scottish Medicines Consortium to recommend their use on the NHS and serving as members of the Managed Access Oversight Group (MAOG) for nusinersen and risdiplam.
Rob Burley, Director of Care, Campaigns and Support at Muscular Dystrophy UK said: “This new data is a vital illustration of the huge progress that has been made in the treatment of SMA and comes as NICE starts the process of making long-term recommendations about access to nusinersen and risdiplam. A key factor in the how much benefit people with SMA get from these new treatments is receiving them as early as possible. We are working as part of the UK SMA Newborn Screening Alliance to secure SMA’s inclusion in the NHS Newborn Screening Programme so that these treatments can be given to people with SMA before symptoms develop and vital muscle function is lost”.