Zolgensma – how it works, is given, and safety considerations – FAQs

Onasemnogene abeparvovec (Zolgensma™) uses harmless, genetically-engineered viruses to increase SMN protein levels. A single injection of Zolgensma into the blood is designed to address the genetic root cause of SMA by replacing the faulty or missing SMN1 gene.

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Zolgensma – the assessment, treatment process and after care – FAQs

With the help of clinical experts, we take you through these questions.

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UK Access Now – England

Infants who have SMA Type 1 and meet eligibility criteria may be assessed for NHS funded treatment with Zolgensma.

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UK Access Now – Scotland

Infants who have SMA Type 1 and meet eligibility criteria may be assessed for NHS funded treatment with Zolgensma.

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UK Access Now – Wales and Northern Ireland

Infants who have SMA Type 1 and meet eligibility criteria may be assessed for NHS funded treatment with Zolgensma.

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Zolgensma Trials and Their Results

Intravenous (IV) Zolgensma has been tested in human clinical trials, with children up to the age of 2 years, since 2014. It has been consistently shown to have a well-understood safety profile and to be well-tolerated in these trials.

When compared to no treatment, zolgensma has been shown to increase the availability of SMN protein in many children. This has led to clinically meaningful improvements in muscle function. Several trials of IV delivered Zolgensma are currently ongoing with a variety of children. This includes those who are older and heavier than in the initial trials

A trial investigating intrathecal (IT) administration of the treatment with older heavier children resumed in August 2021. First results were reported in January 2025

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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. Read about this long journey.

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