About TreatSMA

We are a UK charity made up of people living with spinal muscular atrophy (SMA) and their parents and carers who joined hands to improve the diagnostics, standard of care, social support, and access to treatments in this severe genetic disorder.


We support the efforts to speed up diagnostics and introduce treatment as early as possible. Early introduction of pharmacological therapies offers the highest chance of preventing the disease manifesting itself. We support the introduction of routine newborn screening for SMA as well as wide availability of carrier screening.

Standard of care

TreatSMA aims that hospitals across the country are able to offer the highest standard of medical care to those with SMA. We promote the Standard of Care document and protocols, so as to reduce the risk of severe complications of spinal muscular atrophy and improve the quality of life.

Access to treatments

Everyone with SMA has a right to treatment.

We work to ensure that people with all types of the SMA get the opportunity to access effective treatments. We co-operate with all other stakeholders in the UK SMA community in order to bring about broad access to Spinraza (nusinersen), the first approved treatment for SMA, and all future therapies.

Social support

We advocate that those affected by SMA receive an appropriate level of social support, including access to physiotherapy, hydrotherapy, occupational therapy, supportive tools and equipment – everything that reduces the burden of SMA and makes life with this severe disease more bearable.

How we work

We do not believe that sitting back and waiting or hoping for the government to provide us with everything will get it to our children or ourselves.

Our campaigns highlight the devastating effects that spinal muscular atrophy has on those affected by the disease and shows how the treatment and support can improve their quality of life. For many, it is literally the difference between life and death.

Most of all, we encourage people with SMA, their friends and families to work together and fight together. For the first time ever we have hope! Without everyone getting on board our voices might be lost and the effects of that could be devastating!

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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