The DMD Hub – and gene therapy trials

November 24, 2022 10:15 am

The first boy in the UK to participate in Sarepta’s DMD gene therapy trial enjoyed a starring role in a presentation at the Action Duchenne conference last week.

Charlie is taking part in the EMBARK study, a Phase 3 trial looking at the efficacy and safety of Sarepta’s gene transfer therapy in boys with DMD aged 4.

Previously, clinical trials and studies were often turned away from the UK due to lack of capacity. Since the DMD Hub’s launch, hundreds of boys are now taking part in research across the UK.

DMD Hub’s Emma Heslop told the conference how participant Charlie’s mum Jennifer had filled his details onto the The DMD Hub Central Recruitment Pilot Project. The project aims to create a national contact list of children and adults with DMD who are interested in participating in research studies.

“I’m very glad that the moment I saw the Facebook post about the DMD Hub’s central recruitment project, I thought – I’m going to do that now. I’ve constantly got this great long list of things to do, but without it they would not have had our details, and you would be speaking to some other little boy’s mum or dad – and not me. We would not have been given this potentially life-changing opportunity. We are very, very grateful.”

Find out more about this story and how to take part in clinical research on the DMD Hub’s website:

Alex’s Wish is so incredibly proud to have done our bit with this, we have invested thousands into The DMD Hub and have also support Emma Heslop’s DMD Hub Manager role, thanks to the generosity of our wonderful supporters. This just goes to show the work you do makes such a big difference to families living with Duchenne.

Posted in