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

Nuisnersen (Spinraza™) is an antisense oligonucleotide 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 be have a well-understood safety profile and to be well-tolerated in several different trials across 5q SMA populations.

In many people who have SMA, when compared to placebo or no treatment, nusinersen has been shown to increase the availability of SMN protein, leading to clinically meaningful improvements in muscle function. Several trials of nusinersen are currently ongoing.

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Other published summaries of treatment outcomes

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Our advocacy and progress towards Nusinersen access in the UK

You can read about this long and stressful journey for the SMA Community.

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