New Medicine for DMD, Funded by Joining Jack and Patient Groups

The National Institute for Health and Care Excellence (NICE) has approved vamorolone for use on the NHS to treat Duchenne muscular dystrophy (DMD). Vamorolone, also known by the brand name Agamree, will be available to treat people with DMD aged four years and older.

We have nurtured the development of vamorolone for ten years in a ground-breaking example of venture philanthropy.

Joining Jack, along with our partner charities Duchenne UK and Duchenne Research Fund, invested £750,000 towards a Phase 1 clinical trial in boys with DMD in 2015. We then advised on trial protocol and funded the clinical research site for the vamorolone Phase 2 trial. This then enabled ReveraGen to win a £6 million grant from Europe's Horizon in 2020.

Alex Johnson OBE said: "We were given very little hope and few treatment options when our sons were diagnosed with DMD. We could not and would not accept that and have raised millions of pounds for research into new treatment options for DMD, one of which was vamorolone. We are very proud that a treatment that we have supported for so long will now be available in the NHS."