Risdiplam clinical trials and their results

Researchers have tested risdiplam in human clinical trials since 2015. Risdiplam is not a cure for 5q SMA but in these trials, it has been consistently shown to have an acceptable safety profile and to be well-tolerated. When compared to no treatment, it has led to clinically meaningful improvements in muscle function. Several trials of risdiplam are still ongoing.

Risdiplam works best if started before there is any muscle weakness, or when this is minimal. It is therefore important for treatment to be started as soon as possible.

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What it is, How it works, and Other FAQs

Risdiplam is a small molecule drug that targets the ‘back up’ survival motor neuron 2 (SMN2) gene to produce more SMN protein.

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UK Access Now – England

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

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UK Access Now – Scotland

The NHS in Scotland funds risdiplam.

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UK Access Now – Wales and Northern Ireland

In Wales and Northern Ireland, risdiplam is funded under the same terms as for England’s Managed Access Agreement.

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Our Advocacy and Progress Towards Risdiplam Access 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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