VISION-DMD

Designed to ensure a timely & cost-effective drug development for Duchenne muscular dystrophy

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  • The Project
    • Summary
    • Project Objectives
    • Project Consortium
    • Contact us
  • About DMD
    • What is DMD?
    • Living with Duchenne
    • Current treatment options
    • Duchenne boy’s stories
    • DMD Community
    • Standards of Care for Duchenne muscular dystrophy
  • Study Information
    • What is a clinical study?
    • Phase 2a clinical trial information
    • Phase 2a preliminary study results
    • Phase 2b clinical trial information
    • Phase 2b Preliminary Results
    • MRI – Magnetic Resonance Imaging
  • Drug Development
    • Development of Vamorolone
    • Vamorolone development programme
  • Innovations
    • Return of patient data
    • Bone Health Outcomes
    • Biomarkers
    • Vamorolone study APP for boys
    • Patient Group Funders
    • What is Venture Philanthropy?
  • Resources
    • Relevant publications
      • VISION-DMD INFORMATION APP OVERVIEW
      • VISION-DMD Thought paper
    • VISION-DMD Workshops
      • Workshop series: Standards of Care
      • Workshop series: Biomarkers and Imaging
    • Webinars and presentations
    • Press Releases
    • VISION-DMD project Interviews
      • Collaborating for progress on rare diseases: an interview with Dr. Daria Julkowska, Scientific Coordinator of the European Joint Program on Rare Diseases.
      • Returning vamorolone study data to patient caregivers. What, when and how clinical trial data is returned to study participants. An interview with Dr. Laurie Conklin
      • An Interview with Andrea D’Alessandro – Clinical research project manager at TRiNDS
      • Interview with Jana Haberlová – Paediatric Neurologist at University Hospital Motol
      • Interview with Alex Johnson: a parent and patient foundation funder
    • VISION-DMD Project Newsletters
      • VISION-DMD newsletter sign up
    • VISION-DMD Poster presentations
    • Our privacy policy
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VISION-DMD

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.                                                                  

Project animation produced by World Duchenne Organisation

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement number 667078.

To find out more about the project contact us at [email protected]

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