The National Institute for Health and Care Excellence (NICE) is currently conducting an appraisal of the Duchenne muscular dystrophy treatment vamorolone, a potential alternative to corticosteroids. The appraisal will recommend whether or not the treatment should be made available on the NHS in England. Muscular Dystrophy UK is supporting this process as a ‘Patient Expert’, and we are working to gather the views of the Duchenne community to ensure that these are fully represented in the appraisal process.
NICE are particularly interested in understanding more about the community’s thoughts on existing corticosteroid treatment options, and the potential advantages or disadvantages that vamorolone may have in comparison to them. Help us strengthen our response by answering as many of the questions in this survey as you are able to, and where able please provide examples, as these will help NICE to understand the ‘real-world’ impact Duchenne and of corticosteroids.

Further information about vamorolone is available here.

Thank you for taking part, this survey is anonymous and should take about ten minutes to complete.

The survey will close at midnight on Sunday 4 February 2024.