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

Current treatment options

Currently there is no cure for Duchenne muscular dystrophy. Standards of care include a multi-disciplinary approach, with the input of specialists such as neuromuscular specialists, physiotherapists, occupational therapists, and respiratory and cardiac professionals.

Steroids

There is promising research trying to find a treatment for DMD, however at the moment the only treatment available to all DMD boys is corticosteroids (CS). CS (both prednisone and deflazacort) have been shown to slow the progression of the muscle weakness over a certain period of time and prevent or postpone the development of complications. However, they are associated with many possible side effects, which must be carefully managed. CS associated side effects in DMD include weight gain, behavioural problems, bone fragility, reduced growth and delayed puberty. Other less common side effects are hypertension, glucose intolerance, cataracts and immunosuppression.

In the US, the Food and Drug Administration (FDA) approved Emflaza (Deflazacort) tablets and oral suspension to treat patients age 5 years and older with DMD in February 2017.

Watch the Duchenne Care Videos on Steroids, produced by World Duchenne Organization here

Click here for all World Duchenne Organization Standards of Care videos

Other drugs

Other drugs are beginning to become available for Duchenne muscular dystrophy, including Ataluren (traslarna), which is currently available in some European countries to slow down the progression of symptoms. Ataluren however only works for a small group of boys with DMD who carry a particular mistake in the dystrophin gene (’nonsense’ mutation).

Ataluren, as well as other drugs currently under investigation in clinical trials (utrophin, antisense oligonucleotides, anti-myostatins etc) are meant to be administered in association with CS (they do not replace CS treatment).

Further information about Duchenne muscular dystrophy and treatment can be found on the following websites:

CINRG Research                 Parent Project MD                            Muscular Dystrophy UK Factsheet                         Duchenne UK

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