Cookies on this website
We use cookies to ensure that we give you the best experience on our website. If you click 'Continue' we'll assume that you are happy to receive all cookies and you won't see this message again. Click 'Find out more' for information on how to change your cookie settings.

Screenshot20220426at22.53.49.png

 - 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.