Session eight: Stem cell therapies - towards clinical trials
Chaired by
Hanns Lochmüller - Newcastle University, UK
Francesca Pasinelli - Fondazione Telethon, Italy..
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08:40 - 10:00
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Keynote presentations
- Johnny Huard - University of Pittsburgh, USA
Exhaustion of muscle progenitor cells in muscular dystyrophy: Implication for stem cell therapy
- Chris Denning - Nottingham University, UK
Modelling genetic heart disease using pluripotent stem cells
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10:00 - 10:30
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Panel discussion: stem cell therapies
Moderators : Hanns Lochmüller, Francesca Pasinelli
Panel members : Johnny Huard, Chris Denning
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10:30 - 11:00
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Coffee
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Session nine: What is the future for translational research in rare diseases?
Chaired by
Kate Bushby - Newcastle University, UK
Eric Hoffman - Children's National Medical Center, Washington DC, USA
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11:00 - 12:00
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Keynote speeches
- GertJan van Ommen - Leiden University Medical Center, Netherlands
Fundamental, translational and clinical research for NMDs
- Clemens Müller-Reible - University of Würzburg, Germany
Advances in genome analysis: is more always better?
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12:00 - 12:30
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Panel discussion: access to new therapies and technologies
Moderators: Kate Bushby, Eric Hoffman
Panel members: Gert-Jan van Ommen, Clemens Müller-Reible, Volker Straub
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12:30 - 13:00
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Closing keynote and final remarks
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