VISION-DMD is a collaborative global project aiming 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.
Boys with DMD 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.
This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement number 667078.