Research and access to new treatments - Our impact in 2023

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.

Duchenne UK Impact Report 2023

A landmark year of achievement and impact