What we are working on

What we are campaining to make any and all treatments to SMA available to everyone with the disorder.

In particular, we want to make sure that Spinraza is appraised by NICE and is then offered by the NHS in the entire spectrum of SMA, across all SMA types, ages, and functional abilities.

To this aim, we are reaching out, both as individual families and as a Community, to the decision makers. We tell them what SMA is all about and that even minor functional improvements mean a lot to SMA patients and their carers.

In January 2017, we took part in the Spinraza scoping workshop at NICE, the agency responsible for appraising all treatments in the country. Throughout the winter and spring of 2017, we worked towards making the Spinraza Expanded Access Programme possible in the UK, all whilst the country lagged behind most of the world in starting the programme. We wrote to MPs, we linked up with hospitals across the country, we held meetings with managers and directors at NHS. As a result, and thanks to an involvement of SMA medical experts and other SMA charities, EAP became a reality in the UK this same spring.

In July 2017, we presented the SMA cause at a meeting at the House of Lords.

Throughout the 2018 we engaged in the Spinraza appraisal process, participating in a series of meetings with health authorities in which we always pushed for a wide availability of Spinraza. We also engaged with the MPs as well as reached out to the regulators.

In September, in response to the negative NICE recommendation, we organised protests in London and Manchester.

We are currently putting our our resources to the fight for the only existing effective treatment for SMA.

Latest Posts

  • Calling on all the community to ACT NOW to avoid losing treatments!

    Calling on all the community to ACT NOW to avoid losing treatments!

    Currently, there are two approved treatments for SMA: risdiplam and nusinersen. These treatments were initially approved temporarily under Managed Access Agreements while the NHS and NICE gathered data to assess their cost-effectiveness. Now, as these agreements come to an end, both treatments face a crucial review.

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  • NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam

    NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam

    SMA UK are proud to join together with Treat SMA and MDUK to bring you this webinar about NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam. 2024 sees the end of the managed access agreements (MAAs) for the two treatments. An expert committee will gather to assess the new clinical and real-world evidence, along…

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  • Let the battle begin

    Let the battle begin

    This year the Managed Access Agreement for Spinraza and Risdiplam expires. The MAA was put in place five years ago so the pharmaceutical companies could gather more evidence to resubmit to NICE for approval on the NHS with Biogen extending their MAA by a year.

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