Welcome to the website of VISION-DMD. This collaborative global project aims to develop a safe, effective and affordable therapy for Duchenne Muscular Dystrophy (DMD). This is done by investigating the safety and efficacy of Vamolorone (VBP15) in ambulant DMD boys, and is currently undertaking Phase 2b clinical trial.
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 the potential for reduced side effects. Preclinical studies suggest that vamorolone has the potential to increase the therapeutic window, slow disease progression, and improve quality of life and lifespan for all DMD patients. VISION-DMD is an advanced and innovative clinical programme to develop a safe, effective and affordable therapy for DMD.
Latest update: Santhera Receives Positive CHMP Opinion Recommending Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy Read more here!
Project animation produced by World Duchenne Organisation