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Vamorolone is a potential treatment for inflammation associated with Duchenne muscular dystrophy. It is a potential alternative to currently used corticosteroids and could have less acute side-effects. It is manufactured by the pharmaceutical company Santhera.

How it works

Vamorolone binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. High dose corticosteroids have significant side effects that detract from patient quality of life. Due to Vamorolone’ s potential to ‘dissociate’ efficacy from typical steroid safety concerns, this treatment could be a valuable alternative to corticosteroids. 

What we are doing

NICE is currently conducting an appraisal of the treatment to determine whether or not it should be recommended for use on the NHS. We are takin part in the appraisal process as a formal patient expert. The appraisal is due to conclude in May 2024. 

More information on NICE’s appraisal of Vamorolone can be found here.

Support and information

Duchenne muscular dystrophy

Duchenne muscular dystrophy is a muscle wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys.


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.