VISION-DMD

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

Search

  • 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
  • English
    • Nederlands
    • Français
    • Deutsch
    • Italiano
    • Español

What is DMD?

smiling boy

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 – 5000 live male births, with about 20,000 new cases diagnosed worldwide each year. DMD is caused by a mutation in the gene for dystrophin, which is located on the human X chromosome. This protein is an important component within muscle tissue that provides structural stability to the cell membrane.  It is a progressive disorder that causes muscles to become weaker over time until it affects the whole body. DMD primarily affects boys, the condition in girls tends to be milder, and occurs across all races and cultures. 

Duchenne is often diagnosed between the ages of two and seven. A child with DMD may have difficulty walking, running or jumping and have difficulty standing up. They may learn to speak later than usual, be unable to climb stairs without support and could have behavioural or learning difficulties.

Typically, boys with Duchenne lose their ability to walk between the ages of ten and fourteen. By their late teens, young men lose the strength in their upper bodies, including the ability to move their arms. Also during their teenage years, young men with Duchenne usually need help with breathing at night. Over time, their breathing or respiratory systems weaken, and they require constant support. Young men with Duchenne typically survive into their twenties or early thirties.

Find out more about Duchenne Muscular Dystrophy at the TREAT-NMD website

Follow us on twitter

  • Twitter

EUFlag2020

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 vision-dmd_team@ceratium.eu

VISION-DMD Copyright © 2023 · Log in

Multilingual WordPress with WPML