DMD is an incurable, rare muscle wasting disease. Boys progressively weaken and lose the ability to walk. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. Young men with Duchenne typically live into their late twenties. Corticosteroids are widely recognised to increase muscle strength and delay disease progression but global acceptance as the standard of care is variable due to severe side effects.
Vamorolone is an innovative steroid-like drug designed to retain or improve corticosteroid efficacy and increase membrane stabilization with reduced side effects. Vamorolone aims to increase the therapeutic window, to slow disease progression,n and improve quality of life and lifespan for all DMD patients. This project builds on positive preclinical and Phase 1 results in healthy adult volunteers. Vision-DMD is an advanced and innovative clinical programme to develop a safe, effective and affordable therapy for DMD.