RECENTLY, 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.
Duchenne muscular dystrophy is a serious condition that causes progressive muscle weakness. It affects 2,500 boys and men in the UK and there is no cure for the condition.
Muscular Dystrophy UK, the leading charity for more than 110,000 children and adults in the UK living with one of over 60 muscle wasting and weakening conditions, fully supports the decision. This is a big step forward in the availability of treatment options for Duchenne muscular dystrophy and provides an alternative to the corticosteroids that are currently prescribed.
The charity played a leading role in the SMC’s appraisal of vamorolone, working in partnership with other charities, as well as providing patient expert voices. Gerry McMenemy, from Stirlingshire, shared his son’s experience of living with Duchenne muscular dystrophy following Somhairle’s diagnosis in 2017.
Bringing his lived experience as a parent and ambassador of a child with a muscle-wasting condition Gerry, a member of the Muscular Dystrophy UK Scottish Council and a Trustee of the charity, fully 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.”
Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, also welcomes the news she 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.”
The SMC announcement follows the National Institute for Health and Care Excellence (NICE)’s decision in December 2024 for the drug to become available in England. The NHS in Wales and Northern Ireland generally follow NICE guidance meaning the drug should be available across the whole of the UK.
For more information about Muscular Dystrophy UK visit musculardystrophyuk.org or call our free helpline on 0800 652 6352 (open Mon – Thu 10am – 2pm).
