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 - Spinal muscular atrophy (SMA) is a rare genetic condition that slowly makes muscles weaker over time, causing problems with movement.

- It's caused by a mutation in a gene known as SMN1.

- Zolgensma is an (FDA approved) gene therapy.

- This AAV9 vector delivers a functional copy of the SMN1 gene to the motor neuron cells that need it, stopping symptoms getting worse!




Find out more about the work of the Gill Hyde Gene Medicine research group at the University of Oxford.