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
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Bone Health Outcomes

Bone health and the impact of glucocorticoids on bone is considered one of the highest concerns of patients with DMD and their families. Moderate and high dose corticosteroids are often used to treat conditions with chronic inflammation, including DMD. A common and clinically impactful side effect of corticosteroid treatment is osteoporosis that causes bone fragility, with pharmacological corticosteroid use accounting for over 50% of all cases of bone fragility due to osteoporosis. Both vertebral and long bone fractures can lead to early loss of ambulation and severely impact mobility, while vertebral fractures can be associated with chronic pain.

Bone health outcomes are included in the Phase 2b study as it is important to show vamorolone is less toxic to the skeleton than the high dose glucocorticoid therapy that is used as part of conventional therapy for DMD.

The bone study, in collaboration with Dr. Leanne Ward, Professor of Paediatrics and Research Chair in Paediatric Bone Health at the University of Ottawa, Canada, will evaluate the effect of vamorolone compared to prednisone on bone markers (bone density, bone mass, frequency and severity of vertebral fractures, and frequency of long bone and other fractures).  It will include a central reading of bone investigations including Spinal X-Ray and dual-energy x-ray absorptiometry (DEXA scan). These bone health investigations are recommended as part of international DMD clinical care guidelines for boys with steroid treated DMD and will be part of the outcome measures in the Phase 2b study.

The Ottawa Paediatric Bone Health Research Group will be responsible for the central reading and analysis of lateral spine x-rays (triple read), and lumbar spine and total body bone mineral density (BMD) by DEXA including total body composition analysis in order to evaluate lean muscle and fat mass for this study

For more information on bone health click on the World Duchenne Care video below:

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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 vision-dmd_team@ceratium.eu

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