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

 

Workshops

 

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

Biomarin-logoPTC_logo_tag_4c

Silver Sponsor

sarepta

Bronze Sponsors

Akashi-Logo-280pxpfizer_rgb_pos PrintSanthera PharmaceuticalsSUMMITplc_Logo

Contributors

EBioMedicine logo (1)

Catabasis    Idera_Logo_RGB