Slides and Presentations
The Action Duchenne International Conference 2019 had 43 separate sessions (plus loads of activities for children and teenagers), meaning even those in attendance didn’t get to see everything!
Here we have compiled as many of those presentations as we can, so that the whole international Duchenne community can benefit from them. Please note some of these have been edited (and some we can’t publish at all) as they often contain information and results that have not yet been made available to the wider public.
Developing Effective Treatments for All
Looking at the real world – developing new outcome measures. (Victoria Selby and Laurent Servais)
The age of genomic medicine (Patrick Short)
Gene therapy – the latest updates and a look to the future (Neil Bennett, Jean-Francois Briand and Francesco Muntoni)
Exon skipping technology in Duchenne (Annemieke Aartsma-Rus, Graham McClorey, Aurelie Goyenvalle and Ian Woodcock)
Company updates (Capricor, Catabasis, Italfarmaco, Pfizer, PTC, ReveraGen, Roche, Santhera, Sarepta, Wave)
Action Duchenne update (Neil Bennett)
An update on basic research and why it’s still important (Neil Bennett, Keith Foster and Linda Popplewell)
Company and Researcher Q+A (Capricor, Catabasis, PTC, ReveraGen, Roche, Santhera, Sarepta, Wave)
Developing Effective Treatments for All (continued…)
Stem cell therapies in Duchenne (Neil Bennett, Yung-Yao Lin and Saverio Tedesco)
Clinical trial experiences, considerations and ethics (Bobbie Farsides, Stefan Spinty and Families)
Using existing drugs to treat Duchenne (Steve Winder and Jarod Wong)
Building a Community
Newly diagnosed: Duchenne 101 (Annemieke Aartsma-Rus)
Carriers: genetics and implications (Neil Bennett and Angela Stringer)
Physiotherapy (Marion Main and Jose Longatto)
Talking to your children about Duchenne: diagnosis, treatment, hopes and dreams (David Schonfeld)
Carriers: reproduction (Michela Guglieri)
Learning and behaviour in Duchenne (James Poysky)
Psychology (Gabriella Rands)
A More Inclusive Society
Accessible gaming for Duchenne (Vivek Gohil)
Manual handling (Julie Hewitt and Disability Living Foundation)
Practical inclusion tips for school (Clair Warner)
IEPS and EHCPs (Clair Warner)
Sibling stories (Clare Kassa and siblings)
What to do in an emergency (Jarod Wong and Stefan Spinty)
Air travel with a disability (Chris Wood)
Duchenne education programme (Alban Squires)
Wishes of teens and adults with Duchenne (David Abbott, 2018 presentation)
