What is it, how does it work, how is it given – FAQs

Nuisnersen (Spinraza™) is an antisense oligonucleotide. It is designed to modify the product of the SMN2 “backup” gene to produce more functional SMN protein.

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UK access now – England

Nusinersen is funded by the NHS via a Managed Access Agreement.

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UK access now – Scotland

Nusinersen is funded by the NHS and is available for children. Though there is provision in the NHS agreement for adults, there is no treatment centre in Scotland delivering nusinersen.

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UK access now – Wales and Northern Ireland

Wales and NI follow NICE guidance for England and the guidelines of the Managed Access Agreement.

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Nusinersen trials and their results

Nusinersen has been tested in human clinical trials since 2011. It has been consistently shown to have a well-understood safety profile. It has been well-tolerated in several different trials across 5q SMA populations.

When compared to placebo or no treatment, nusinersen has been shown to increase the availability of SMN protein in many people. This has led to clinically meaningful improvements in muscle function. Several trials of nusinersen are still ongoing.

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Other published summaries of treatment outcomes

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Our advocacy and progress towards access to Nusinersen in the UK

We have worked hard with the SMA Community, Muscular Dystrophy UK, TreatSMA and clinicians to advocate for access, and will continue to work towards access throughout the UK.

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