For more than a decade, Duchenne UK has been driving pioneering research into treatments for Duchenne muscular dystrophy (DMD). This year saw a breakthrough; the regulatory approval of the first ever treatment for all people with DMD, Vamorolone.
We are proud to have contributed to this milestone in making new treatment options available to people living with DMD.
And we are proud of the work our DMD Hub continues to drive to enable access to treatments.
A global trial, which was run in the UK through our DMD Hub, showed Givinostat, another drug seeking regulatory approval in DMD, can slow disease progression and delay loss of ambulation.
Our DMD Hub's network now has DMD trial sites in Alder Hey, Birmingham, Bristol, Glasgow, Leeds, London (Evelina), London (GOSH), Manchester, Newcastle, Oswestry, and Oxford, which have been undertaking trials for people with DMD from across the UK in 2023. By the end of 2023, the DMD Hub has run 41 trials and 574 people with DMD have taken part in a DMD Hub trial since it was set up in 2016.
Our extensive portfolio of research projects that we funded in 2023 includes research into stem cells and their ability to regenerate muscles in DMD, developing new electronic clinical outcome assessments that can be used in DMD clinical trials, and a research programme on nutrition and weight management in boys living with DMD.
Our DMDhome digital platform that we are working on with Aparito has entered the validation phase. DMDhome uses video capture and computer vision analysis to measure limb function in DMD. The study will aim to validate this new approach for use in clinical trials and potentially in the approval of new medicines for DMD.
In 2023, our Project HERCULES helped to support health technology assessments of new treatments for DMD such as Vamorolone, which is currently being assessed by NICE.
A landmark year of achievement and impact