Action Duchenne International Conference 2016 Agenda

Below are the presentation slides from the 14th Action Duchenne International Annual Conference, which took place on Friday 11 and Saturday 12 November. The presentations are split into the following four tables: Friday AM, Friday PM, Saturday AM and Saturday PM. Please note not all speakers are able to provide their presentations for public use.

Photo by

Friday 11 November AM Sessions

9.15-9.55 9.55-10.45 11.00-11.50 11.50-12.40
Plenary Sessions
Kings Suite
Living well and independently
- Ros Quinlivan
Current clinical trial updates -
Cardiac (heart) research) -
John Bourke
Cognitive research and behaviour - Veronica Hinton and Valeria Ricotti
DMD Pathfinders Monarch Suite Teenage to adult transition (the care aspect) - Assistive services summary and demonstration
Workshop One Supporting children and families -
David Schonfeld
Physiotherapy tips - Marion Main
Target Roundtables Inflammation - Michela Guglieri Hormones and puberty - Tim Cheetham Muscle structure and cell biology - Karl Bettelheim
Workshop Three Overview of clinical trial process - Jordan Butler and Katie Groves The ins and outs of taking part in clinical trials - Alex Smith, Alexandra Johnson and Bernie Mooney and Kate Maurice A guide to Duchenne genetics - Jo McCauley Carrier genetics and support -
Sue Kenwrick

Photo by

Friday 11 November PM Sessions

  14.10-15.30 15.40-16.30 16.30-17.30
Plenary Sessions Kings Suite Access to medicines panel session - Natalie Bere (EMA), Daniel O'Connor (EAMS) and Paul Schofield (ABPI), Edmund Jessop and Sheela Upadhyaya New generation exon skipping updates - Matthew Wood and Aurelie Goyenvalle How to have the best transition to adulthood: lessons from the Takin' Charge project
DMD Pathfinders Monarch Suite Adult health and well-being - Marina Di Marco, Jon Hastie and Mark Chapman Sex and relationships - Maddie Blackburn Informing children and collecting data: a live clinical trial app demo - Olav Veldhuizen and Cathy Turner
Workshop One Education Health and Care Plans: getting the right support - Janet Hoskin and Nick Catlin
Target Roundtables Calcuim/sodium exchange - Florence Porte Rare repurposing in Duchenne -
Steve Winder
Workshop Three Housing adaptations, your rights and obtaining benefits - Robbie Spence

Photo by

Saturday 12 November AM Sessions

  9.00-10.00 10.00-11.00 11.15-12.15 12.15-13.15
Plenary Sessions Kings Suite Promising new research - Dominic Wells and Stanley Froehner Overview of new trials - Thomas Voit Natural History data: everything you need to know - Eugenio Mercuri Outcome measures in trials - Francesco Muntoni
DMD Pathfinders Monarch Suite Physiotherapy tips - Marion Main, Jordan Butler and Marina Di Marco Respiratory research - Anita Simonds Preventative care (nutrition and ventilation) - Anita Simonds, Jon Hastie and Mark Chapman
Workshop One What to do in an emergency? -
Michela Guglieri
Standards of care (ages 5-10) -
Michela Guglieri
Standards of care (ages 11-16) - Michela Guglieri
Target Roundtables Other protein targets - Kay Davies Exon skipping - Annemieke Aartsma-Rus Gene therapy - George Dickson
Workshop Three Duchenne from the beginning -
Annemieke Aartsma-Rus
Supporting children and families -
David Schonfeld

Photo by

Saturday 12 November PM Sessions

  14.15-15.15 15.15-16.15 16.15-17.00
Plenary Sessions Kings Suite Genetic replacement technology - Jeff Chamberlain, George Dickson and Ronald Cohn Other mutations and protein targets - Kay Davies Where are we and what have we learned from recent trials?
Francesco Muntoni
Thomas Voit
DMD Pathfinders Monarch Suite Respiratory focus group and practical tips - Anita Simonds, Jon Hastie and Mark Chapman Having carers that are not your parents - Mark Chapman, Jon Hastie, Farhan Mian and Ravi Mehta
Target Roundtables Stem cell therapy - Jenny Morgan Muscle cellular stress - Keith Foster
Workshop Three Support for learning and behaviour at school and home - Janet Hoskin and Nick Catlin Wheelchair access and how to get other services - Dean Jose and Angela Stringer

Photo by

As an extra session we also had the fantastic Virginia Arechavala Gomeza PhD, present the World Muscle Society Conference highlights.

If you would like more information about the presentations or have any questions please contact [email protected]