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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Risdiplam Clinical Trials and Their Results

Risdiplam has been tested in human clinical trials since 2015. It has been consistently shown to 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, risdiplam has been shown to increase the availability of SMN protein, leading to clinically meaningful improvements in muscle function. Several trials of risdiplam are currently ongoing.

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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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