2013 International Conference on DMD


The CARE-NMD International Conference on DMD took place at the Hilton Westend, Budapest on 18th-19th April 2013.

Approximately 125 attendees from around the world took part, including representatives of patient organisations, academic and clinical experts, and policymakers.

We are pleased to make available presentations from the conference available below, where we have permission to do so.

Download the Conference Programme

Thursday 18th April

Session 1 Introduction to DMD and DMD Care

Session 2 Evaluating Care for DMD

Session 3 Quality of Life in DMD

Session 4 Current Research 1: Answering Open Questions in the Care Standards

Friday 19th April

Session 5 Current Research 2: Future Therapies and Regulatory Perspectives

  • Mutation‐specific approaches for treatment in DMD - Nathalie Goemans, Universite Catholique de Louvain
  • Stem cells and gene therapy: hype versus reality - Thomas Voit, Institut de Myologie
  • Implementing new treatments in rare diseases: the Pompe experience - Ria Broekgaarden, Vereniging Spierziekten Nederland
  • Regulatory perspectives on DMD therapies - Bruno Sepodes, European Medicines Agency
  • Panel discussion/Q&A - All participants

Session 6: An International Perspective: Networks and the Future of DMD Care


    Organised by the CARE-NMD Consortium
    Project Leader PD Janbernd Kirschner

    CARE-NMD International Conference on DMD

    We are delighted to announce a major international conference on Duchenne muscular dystrophy (DMD) on 18th-19th April 2013 at the Hilton Westend in Budapest.