Searching for a cure, Improving lives

Presentation Slides from Conference 2016

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.

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Friday 11 November – AM Sessions

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 SuiteTeenage to adult transition (the care aspect) - Assistive services summary and demonstration
Workshop OneSupporting children and families -
David Schonfeld
Physiotherapy tips - Marion Main
Target RoundtablesInflammation - Michela GuglieriHormones and puberty - Tim CheethamMuscle structure and cell biology - Karl Bettelheim
Workshop Three Overview of clinical trial process - Jordan Butler and Katie GrovesThe 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 McCauleyCarrier genetics and support -
Sue Kenwrick

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Friday 11 November – PM Sessions

Plenary Sessions Kings SuiteAccess to medicines panel session - Natalie Bere (EMA), Daniel O'Connor (EAMS) and Paul Schofield (ABPI), Edmund Jessop and Sheela UpadhyayaNew generation exon skipping updates - Matthew Wood and Aurelie GoyenvalleHow to have the best transition to adulthood: lessons from the Takin' Charge project
DMD Pathfinders Monarch SuiteAdult health and well-being - Marina Di Marco, Jon Hastie and Mark ChapmanSex and relationships - Maddie BlackburnInforming children and collecting data: a live clinical trial app demo - Olav Veldhuizen and Cathy Turner
Workshop OneEducation Health and Care Plans: getting the right support - Janet Hoskin and Nick Catlin
Target RoundtablesCalcuim/sodium exchange - Florence PorteRare repurposing in Duchenne -
Steve Winder
Workshop ThreeHousing adaptations, your rights and obtaining benefits - Robbie Spence

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Saturday 12 November – AM Sessions

Plenary Sessions Kings SuitePromising new research - Dominic Wells and Stanley FroehnerOverview of new trials - Thomas VoitNatural History data: everything you need to know - Eugenio MercuriOutcome measures in trials - Francesco Muntoni
DMD Pathfinders Monarch SuitePhysiotherapy tips - Marion Main, Jordan Butler and Marina Di MarcoRespiratory research - Anita SimondsPreventative care (nutrition and ventilation) - Anita Simonds, Jon Hastie and Mark Chapman
Workshop OneWhat to do in an emergency? -
Michela Guglieri
Standards of care (ages 5-10) -
Michela Guglieri
Standards of care (ages 11-16) - Michela Guglieri
Target RoundtablesOther protein targets - Kay DaviesExon skipping - Annemieke Aartsma-RusGene therapy - George Dickson
Workshop ThreeDuchenne from the beginning -
Annemieke Aartsma-Rus
Supporting children and families -
David Schonfeld

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Saturday 12 November – PM Sessions

Plenary Sessions Kings SuiteGenetic replacement technology - Jeff Chamberlain, George Dickson and Ronald CohnOther mutations and protein targets - Kay DaviesWhere are we and what have we learned from recent trials?
Francesco Muntoni
Thomas Voit
DMD Pathfinders Monarch SuiteRespiratory focus group and practical tips - Anita Simonds, Jon Hastie and Mark ChapmanHaving carers that are not your parents - Mark Chapman, Jon Hastie, Farhan Mian and Ravi Mehta
Target RoundtablesStem cell therapy - Jenny MorganMuscle cellular stress - Keith Foster
Workshop ThreeSupport for learning and behaviour at school and home - Janet Hoskin and Nick CatlinWheelchair access and how to get other services - Dean Jose and Angela Stringer

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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


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