Searching for a cure, Improving lives

Presentation Slides Conference 2015

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Friday 6 November 2015


King’s Suite


International conversation: living as adults with Duchenne


Mark Chapman and Jon Hastie – DMD Pathfinders


Anny Madsen – Rehabiliterings Center For Muskelsvind


Promising New Drug Candidates


Promising new drug candidates for DMD – Dominic Wells – Royal Veterinary College London


Phytocannabinoids and Muscle – Keith Foster – University of Reading


Exon Skipping – Where are we?


Ed Kaye – Sarepta


Corinne Betts – University of Oxford


Aurelie Goyenvalle – University of Versailles


Other strategies for different mutations


Stuart Peltz – PTC Therapeutics


Nicolas Wein – Nationwide Children’s Hospital , Columbus , Ohio


Charles Gersbach – Duke University


Best preventative actions in cardiac and respiratory 


John Bourke – Freeman Hospital and Newcastle University


Pradeep Mammen – UT Southwestern Medical Centre


Adult Duchenne Research


Ros Quinlivan – National Hospital Queens Square


Saturday 7 November



Gene Therapy – Progress in the clinic




Targeting Related Proteins


Givinostat – Paolo Bettica – Italfarmaco SpA


Myostatin – Michael Binks – Pfizer


Utrophin – Jon Tinsley – Summit PLC


TVN-102 (recombinant biglycan) – Justin Fallon – Tivorsan Pharmaceuticals


Advances in Biomarkers and Clinical Outcome Measures


AD biomarker introduction- Annemieke Aartsma-Rus – Leiden University Medical Centre


Inflamation and Fibrosis 


Joanne Donovan – Catabasis Pharmacueticals


Diana Escolar – Akashi Therapeutics


Inflammation in DMD VBP15 – Michela Guglieri – JWMDRC Newcastle University


Access to drugs : reducing bottlenecks


Matt Cooper – NIHR Clinical Research Network


David Cavalla – Healx


Nick Meade – Genetic Alliance UK


Orphan Designation and Incentives Europe – Segundo Mariz – EMA


Patient Values and Preferences: Methods for Obtaining and Assessing Voices of Patients – Pat Furlong – PPMD


Elizabeth Vroom- UPPMD




Explaining Therapies – Annemieke Aartsma-Rus – Leiden University


Important Points About Molecular Biology and Genetics in Relation to DMD – Karl Bettleheim – Formerly Microbiological Diagnostic Unit Public Health Laboratory Melbourne, Australia


Steroids in Duchenne Muscular Dystrophy – Henriette van Ruiten – The John Walton Muscular Dystrophy Research Centre


Non-Ambulatory Outcome Measures – Anna Mayhew -The John Walton Muscular Dystrophy Research Centre


Genetic Modifiers – Pietro Spitali – Leiden University Medical Centre


Biomarkers – Alessandra Ferlini – Universita de Ferrara


Combination Therapy – Dominic Wells – Royal Veterinary College , University of London


Nutrition and Dietary Advice – Angela Reddy – King’s College , University of London


Standards of Care Post Diagnosis – Michela Guglieri – JWMDRC Newcastle University




With thanks to the sponsors and contributors who helped make our 2015 conference  accessible to our community

Platinum Sponsors



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