New drug for Duchenne muscular dystrophy to be made available in Scotland
13 January 2025
Today (Monday 13 January) the Scottish Medicines Consortium (SMC) has accepted the drug vamorolone (also sold under the brand name Agamree) as a treatment option for people aged four years and over in Scotland with Duchenne muscular dystrophy.
We’re delighted to have played a leading role in the SMC’s appraisal of vamorolone, working in partnership with other charities, as well as providing patient expert voices.
A welcomed decision
Gerry McMenemy, Muscular Dystrophy UK Scottish Council member and a Trustee, shared his son’s experience of living with the condition following Somhairle’s diagnosis in 2017.
Bringing his lived experience as a parent and ambassador of a child with a muscle wasting condition Gerry, he welcomes the news. He said: “This is a huge moment for families living with Duchenne muscular dystrophy, and indeed Muscular Dystrophy UK and the wider muscle wasting and weakening community. As a parent with a child with a neuromuscular condition, you are constantly battling for their needs to help give them the best quality of life possible, whether it’s their care, accessibility or access to treatments.”
“This announcement will make a significant difference to those living with Duchenne in Scotland and their families.”
Gerry McMenemy
Muscular Dystrophy UK Scottish Council member and a Trustee
Our Director of Research and Innovation, Dr Kate Adcock said: “We’re proud to have played a key role in ensuring vamorolone is available for children with Duchenne through NHS Scotland. Throughout the appraisal process, we heard first hand about the dilemma that faces families when it is recommended that their child starts taking corticosteroids. The associated side effects, including weight gain, bone weakness and mood changes, cause huge concern and the community shared powerful testimony about their experiences and the benefit that an alternative treatment option would bring.”
“To now have the treatment available is a great step forward and potentially life changing.”
Dr Kate Adcock
Director of Research and Innovation, Muscular Dystrophy UK
Next steps
Following announcements by the SMC and NICE, the drug should now become available across the whole of the UK as the NHS in Wales and Northern Ireland generally follow NICE guidance.
It will be up to each individual NHS Health Board in Scotland to declare when they will make the medicine available.
If you have any questions about eligibility for the treatment, speak with your clinical team.