Zolgensma clinical trials and their results

Researchers have tested intravenous (IV) Zolgensma in human clinical trials with children up to the age of 2 years since 2014. Zolgensma 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. Trials with older heavier children are ongoing. These include giving Zolgensma via a lumber puncture (intrathecal (IT) administration).

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Read more about Zolgensma and other treatments in the links below:

Access for children – England, Wales, Northern Ireland

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Access for children – Scotland

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Our advocacy and progress towards Zolgensma access in the UK

We have worked hard with the SMA Community, MDUK, TreatSMA and clinicians, to advocate for access.

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