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Zolgensma (also known as onasemnogene abeparvovec) is the gene therapy for SMA made by Novartis Gene Therapy .

How does it work?

It addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells. This gene is critical for the function of the nerves that control muscles. 


It is available in England, Wales and Northern Ireland for the treatment of both symptomatic and pre-symptomatic SMA in babies. 

More information on NICE’s guidance for symptomatic use can be found here and for pre-symptomatic use here.

Zolgensma was accepted for restricted use within NHS Scotland by the Scottish Medicines Consortium in March 2021. More information about its assessment can be found here.

Support and information

Spinal muscular atrophy

Spinal Muscular Atrophy (SMA) is a rare, genetically inherited neuromuscular condition. It causes progressive muscle weakness and loss of movement due to muscle wasting (atrophy). This may affect crawling and walking ability, arm, hand, head and neck movement, breathing and swallowing.


We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.

Our support services

We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.

Our helpline is open Monday-Thursday 10am-2pm.