Session five: Emerging technologies and SOPs in diagnostics and biomarkers
Chaired by
Nicolas Lévy - Faculté de Medecine de la Timone, Marseille, France
Alessandra Ferlini - University of Ferrara, Italy
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08:40 - 10:00
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Keynote presentations
- Nicolas Lévy - Faculté de Medecine de la Timone, Marseille, France
High throughput molecular screening for neuromuscular disorders - the NMD-Chip consortium project
- Alessandra Ferlini - University of Ferrara, Italy
Biomarkers discovery in neuromuscular diseases by omic procedures
- Eric Hoffman - Children's National Medical Center, Washington DC, USA
New approaches to diagnostics and biomarkers
- Giuseppe Novelli - University of Rome Tor Vergata, Italy
Clinical Utility and Validity of Genomic Biomarkers
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10:00 - 10:30
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Panel discussion: sharing resources
Moderators: Nicolas Lévy, Alessandra Ferlini
Panel members: Eric Hoffman, Giuseppe Novelli
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10:30 - 11:00
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Coffee
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Session six: Antisense technologies - strategies and successes
Chaired by
Steve Wilton - University of Western Australia, Australia
Annemieke Aartsma-Rus - Leiden University Medical Center, The Netherlands
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11:00 - 12:00
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Keynote presentations
- Francesco Muntoni - Institute of Child Health, University College London, UK
Lessons on Development of AON Drugs for Duchenne Muscular Dystrophy
- Nathalie Goemans - University Hospitals, Leuven, Belgium
Exon skipping with 2OMePS antisense oligonucleotides in DMD : current clinical trials and future perspectives
- Kathie Bishop - Isis Pharmaceuticals, USA
Development of Antisense Oligonucleotide Therapeutics for the Treatment of SMA and DM1
- Aurelie Goyenvalle - Oxford University, UK
AAV-U7snRNA mediated Exon-skipping Approach for Duchenne Muscular Dystrophy Therapy
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12:00 - 12:30
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Panel discussion: Expectations versus reality
Moderators: Steve Wilton, Annemieke Aartsma-Rus
Panel members: Francesco Muntoni, Nathalie Goemans, Kathie Bishop,
Aurelie Goyenvalle
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12:30 - 13:30
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Lunch
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13:30 - 15:30
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Poster session
Posters to be presented: PCR1 to PCR22; REG1 to REG9; SEI1 to SEI9
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15:30 - 16:00
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Coffee
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16:00 - 16:40
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Keynote presentation
- Ed Connor - Children's National Medical Center, Washington DC, USA
Muscular Dystrophy and drug development - challenges and opportunities
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Session seven: Life Quality versus Quality of Life
Chaired by
Pauline McCormack - PEALS, Newcastle University, UK
Thomas Sejersen - Karolinska Institute, Stockholm, Sweden
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16:40 - 17:00
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Q&A session: life quality versus quality of life
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17:00 - 17:45
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Keynote presentations
- Tom Shakespeare - World Health Organisation
Disabling barriers: break to include
- Gail Geller - Johns Hopkins University, Baltimore, USA
The paradox of promise and the many faces of hope in Duchenne muscular dystrophy
- Patrick Moeschen - Independent speaker, USA (via video-link)
Not waiting to live, and not living to wait. How I am beating MD
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17:45 - 18:30
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Panel discussion: The realities of participation in clinical studies
Pauline McCormack, Thomas Sejersen, Tom Shakespeare,
Gail Geller, Cynda Rushton (Johns Hopkins), Pat Moeschen, Robert Palm (Progena Foundation)
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Conference Gala Dinner (for all delegates)
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20:00 - 23:00
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