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


Inflammation 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


Silver Sponsor


Bronze Sponsors

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