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

• Welcome - Jan Kirschner, Universitätsklinikum Freiburg & Veronika Karcagi, Hungarian National Institute of Environmental Health
• Overview of the field - Kate Bushby, Newcastle University
• A Global perspective on DMD care and training activities - Andoni Urtizberea, Hôpital Marin de Hendaye
• Results of the CARE‐NMD questionnaire - Jan Kirschner, Universitätsklinikum Freiburg

Session 2 Evaluating Care for DMD

• The CARE‐NMD survey in Japan - En Kimura, Japanese National Center of Neurology & Psychiatry
• The Duchenne Connect survey in the USA - Holly Peay, Parent Project Muscular Dystrophy
• The Australian approach to surveying care - Hugh Dawkins, Government of Western Australia
• Panel discussion/Q&A - All participants

Session 3 Quality of Life in DMD

• CARE‐NMD quality of life questionnaire results - Jes Rahbek, Birgit Steffensen, RehabiliteringsCenter for Muskelsvind
• Living with DMD: patient and family perspectives - Peter Mikkelsen, Denmark; Gergely Bujdosó, Hungary
• The psychology of Duchenne Muscular Dystrophy - Jos Hendriksen, Kempenhaeghe expertise centre for epileptology, sleep medicine and neurocognition
• The role of patient organisations - Elizabeth Vroom, United Parent Project Muscular Dystrophy; Jes Rahbek, RehabiliteringsCenter for Muskelsvind
• Panel discussion/Q&A - All participants

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

• Newborn Screening in DMD: Outcomes of the ENMC Workshop - Elizabeth Vroom, United Parent Project Muscular Dystrophy
• Cardiac Treatment for DMD - John Bourke, Newcastle University
• Steroid use: current recommendations and future research - Ulrike Schara, Universitätsklinikum Essen
• Open issues in the Standards of Care and further developments - Thomas Sejersen, Karolinska Institutet
• Difficulties in the transition from childhood to adulthood - Arpad von Moers, DRK‐Kliniken Berlin
• Panel discussion/Q&A - All participants

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

• International networks and DMD registries - Hanns Lochmüller
• National genetic registries: the Australian experience - Hugh Dawkins, Government of Western Australia
• National genetic registries: the Hungarian experience - Veronika Karcagi, Hungarian National Institute of Environmental Health
• The future of DMD care and concluding remarks - Jan Kirschner, Universitätsklinikum Freiburg

 

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