Welcome to the website of Vision-DMD. This collaborative global project aims to develop a safe, effective and affordable therapy for Duchenne Muscular Dystrophy. This is done by investigating the safety and efficacy of Vamolorone (VBP15)in ambulant Duchenne Muscular Dystrophy (DMD) boys, and is currently undertaking Phase 2a and 2b clinical trials.
DMD is an incurable, rare muscle wasting disease. Boys progressively weaken and lose the ability to walk. This 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, delay disease progression and are part of the International Standards of Care, but also come with side effects which can vary form very mild to more severe.
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, 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.