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Presentation Slides from Conference 2017

Action Duchenne International Conference 2017 Agenda

Below are the presentation slides from the 15th Action Duchenne International Annual Conference, which took place on Friday 10 to Sunday 12 November. The presentations are split into the following four tables: Friday AM/Friday PM, Saturday AM/Saturday PM and Sunday AM/Sunday PM

Please note not all speakers are able to provide their presentations for public use.


Friday 10 November – AM Sessions

Plenary Sessions
Queens Suite
Adults with DMD (Hastie, Chapman, Wright)Genetic Modifiers and molecular biomarkers (Spitali & Roberts)Animal models in Duchenne and Becker (Aartsma-Rus)Outcome measures for Duchenne (Domingos)
DMD Pathfinders Arden RoomNorth Star assessment explained (di Marco)Personalisation in neuromuscular services (Austin & Newton)Assistive technology marketplace
Parallel Session 1Duchenne 101: from the beginning (Aartsma-Rus)Practical physiotherapy tips (Main)
Parallel Session 2Newly diagnosed support for families (Poysky & Action Duchenne Trustees)Carrier information and support (Kenwrick, Bowns & Hewitt)
Teenagers Birmingham WellbeingAccesible Multi Sports (Salmon & Team)


Friday 10 November – PM Sessions

Plenary Sessions
Queens Suite
Recent developments in the DMD/BMD field (Voit)Duchenne and Becker TACT updates (Wagner)Late pre-clinical updates: fast track to the clinic (Dorchies & Fischer)Health technology assessments in detail (Marshall)
DMD Pathfinders Arden RoomNutrition, swallowing & feeding tubes (Hastie, Allen & Hawthorn)Holidaying as an adult with Duchenne (Chapman, Wright, Ashby & Drummond)Sex & Relationships (Hastie, de Than & McGinlay)
Parallel Session 1What to do in an emergency (Van Ruiten)Steroid information & management (Guglieri)Is there a future for cell therapy? (Morgan)DMD Registry Steering Committee
Parallel Session 2Applying research to adults roundtable workshop (Quinlivan & Mayhew)Exon skipping research updates (Wood)Electronic wearable devices for Duchenne (Seferian & Faisal)
Teenagers Special effect gaming (Gohil & Messenger)CAD/3D Printing (Burton & Warwick University)


Saturday 11 November – AM Sessions

Plenary Sessions
Queens Suite
Nutritional challenges in Duchenne (Mochrie)Heart research & updates (Bourke & Hor)Lung research & updates (Shields)Brain research (Doorenweerd)
DMD Pathfinders Arden RoomHow to get a good night's sleep & positioning (Brown)Physio for adults (di Marco)Employment & volunteering with DMD (Hastie, Eichner, James & Lanham)Managing a care team (Chapman, Hastie, Gohil & Hawthorn)
Parallel Session 1Behaviour/coping strategies & support (Poysky & Action Duchenne Trustees)Updated standards of care in detail (Guglieri)
Parallel Session 2Becker muscular dystrophy clinical updatesBMD research updatesBMD industry discussion
Teenagers Birmingham WellbeingTheatre workshop (Birmingham Conservatoire)


Becker Muscular Dystrophy Workshop

BMD - transition to adult care / overall caring in BMDCardiac BMD care & managementPhysiotherapy practical tipsBecker natural history updateQuestion & Answer sessionCommunity panel session
Kathryn WagnerKan HorMarion MainPaula Clemens & Kan HorItalfarmaco, Summit & other industry panelistsAlejandra Pareda, Jon Powton & other BMD adults




Saturday 11 November – PM Sessions

Plenary Sessions
Queens Suite
Early clinical trials (Voit)Gene therapy panel session (Dickson, Duan & Cohn)Beginner's guide to UK treatment reimbursement (Marshall)
DMD Pathfinders Arden RoomAnxiety, depression & well-being (Chapman, Poysky & di Marco)Rising above Duchenne (Culhane & Hastie)Adult ventilation: from NIV to trachy (Hastie, Hawthorn & Chapman)
Parallel Session 1Other potential drug targets in Duchenne (Davies)Bone research & updates (Wong)Late clinical trial updates (Majumdar)Gene therapy: the basics (Yanez-Munoz)
Parallel Session 2Info & tips on adrenal insufficiency and emergency are plans (Dunne)Education, health & care plans (Catlin & Hoskin)Housing adaptations (Smith, Bloor & Stringer)
Teenagers Theatre workshop (Birmingham Conservatoire)Zumbalates (Main)


Adult Symposium

Overview of paediatric trials with non-ambulatory armsAdult standards of care & North Star NewtworkNon-ambulatory outcome measures & physiotherapy trainingAdults viewpoint in participating in clinical trialsQuestion & Answer sessionDiscussion with healthcare & industry colleagues
Thomas VoitRos QuinlivanAnna MayhewJon HastieAdrian Warnock




Sunday 12 November

Plenary Sessions
Queens Suite
Learning & behaviour of Duchenne (Poysky & Colvin)Practical tips for families: how to get the info & services you need (Bott, White, Griffin & Dheensa)Ethics & research: the basics (Samuel)Summary & farewell
Parallel Session 1Company summary presentations (Capricor, Catabasis, Italfarmaco, NS Pharma, Pfizer, PTC...cont)Company Q+A panel session (cont...ReveraGen, Roche, Santhera, Sarepta, Solid, Summit & WAVE)
Parallel Session 2Outcome measure updates (Majumdar)Analysis of Duchenne natural history data to accelerate clinical trials (Signorovitch & Larkindale)

If you would like more information about the presentations or have any questions please contact


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