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

 

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

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Contributors

EBioMedicine logo (1)

Catabasis    Idera_Logo_RGB 



 

 

 

 

 

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