Skip to content

NICE starts appraisal process for Duchenne muscular dystrophy treatment vamorolone

18 August 2023

On 1 August 2023, the National Institute of Health and Care Excellence (NICE) formally launched its appraisal of the Duchenne muscular dystrophy treatment vamorolone. It has been created as a potential alternative to steroid treatment, also known as corticosteroids or glucocorticoids. Steroid treatment is a routine part of treatment for Duchenne as it can improve muscle strength; however, it can have significant side effects that reduce the quality of life for those taking it. 

We are partnering with Action Duchenne and Duchenne UK to make sure the views of the Duchenne community are heard by NICE in the appraisal process, scheduled to take place on 5 March 2024. 

How you can share your views 

  • Along with our partners, we are surveying people with Duchenne muscular dystrophy and their families about their experience of steroid treatment and of vamorolone, should you have been part of vamorolone treatment trials. The information gathered will be used in the submission of written evidence to NICE by patient organisations. The survey is open until 17 September. It is an anonymous survey and will take you around ten minutes to complete. We are not gathering any personal data that can identify you or be used to contact you. 
  • Take part in our survey
  • NICE is also looking for people with experience of receiving vamorolone to speak at the appraisal meeting in March. If this is something that you would be interested in doing, please contact by Wednesday 23 August.

Join the Muscular Dystrophy UK community

Share your experience and find advice from others with similar conditions.