Our 2019 programme has now been finalised and can be found below. The PDF version is available here.
Monday 9th December 2019
| 17:00 | - | 19:00 | Early Registration
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| 18:00 | - | 19:30 | Drinks Reception
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Tuesday 10th December 2019
| 8:00 | - | 9:00 | Registration
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| 9:00 | - | 9:15 | Welcome address
Chaired by :
Annemieke Aartsma-Rus – Leiden University Medical Center, Netherlands
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| 9:15 | - | 10:15 | Keynote Lecture
PresentationsWhen being a scientist is not enough Mayana Zatz - Professor of Genetics, University of São Paulo, Brazil |
| 10:15 | - | 10:45 | Coffee break
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| 10:45 | - | 12:15 | Patient engagement
Chaired by :
Fabiola Bertinotti – Patient representative, Italy
Ria Broekgaarden – Vereniging Spierziekten Nederland (Dutch Neuro Muscular Disease Patientorganization), The Netherlands
PresentationsThis session with close with a panel discussion and questions. |
| 12:15 | - | 14:00 | Lunch break
PresentationsLunchtime Symposia – Serepta - , Lunchtime Symposia – Biogen’s vision to transform the therapeutic landscape of neuromuscular diseases - , |
| 14:00 | - | 15:30 | Adults with progressive neuromuscular disease
Chaired by :
Anri Human – Sefako Makgatho Health Sciences University, South Africa
Zohar Argov – Hadassah University Hospital, Israel
PresentationsNutritional assessment and interventions in adult NMD Heidi Zweers - Radbound University Nijmegen Medical Centre, NetherlandsExercise in neuromuscular disease: a moving topic Nicole Voet - Radbound University Medical Center, NetherlandsThis session with close with a panel discussion and questions. |
| 15:30 | - | 16:00 | Coffee break & posters
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| 16:00 | - | 17:30 | New technologies & platforms
Chaired by :
Jordi Díaz-Manera – Hospital de la Santa Creu I Sant Pau, Barcelona, Spain
Volker Straub – John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
PresentationsDeep learning and imaging recognition Jordi Díaz-Manera - Hospital de la Santa Creu I Sant Pau, Barcelona, SpainSupporting the collection of post-marketing surveillance data Craig Campbell - M.D. Head Division of Pediatric Neurology and TGDOC Chair, Western University, CanadaThe EURO-NMD Clinical Patient Management System (CPMS) Teresinha Evangelista - Researcher and EURO-NMD Coordinator, INSERM Institute of myology, FranceThis session will close with a panel discussion and questions |
| 19:30 | - | 23:00 | Networking dinner
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Wednesday 11th December 2019
| 9:00 | - | 10:30 | Personalised medicine
Chaired by :
Annemieke Aartsma-Rus – Leiden University Medical Center, Netherlands
Volker Straub – John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
PresentationsCase study of personalized medicine - , CRISPR-Based Genome Editing for Duchenne Muscular Dystrophy – Successes Charles Gersbach - Duke University, Durham NC, United StatesCRISPR-Based Genome Editing for Duchenne Muscular Dystrophy – Challenges Annemieke Aartsma-Rus - Leiden University Medical Center, NetherlandsThis session will close with a panel discussion and questions |
| 10:30 | - | 11:00 | Coffee break
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| 11:00 | - | 12:30 | Access to drug treatment
Chaired by :
Ans van der Ploeg – Netherlands
Ria Broekgaarden – Vereniging Spierziekten Nederland (Dutch Neuro Muscular Disease Patientorganization), The Netherlands
PresentationsEURORDIS vision and proposals to address current challenges of access to new treatments Yann Le Cam - Chief Executive Office, EURORDIS - Rare Diseases Europe, FrancePatient and clinician perpective on access to therapy Ria Broekgaarden - Vereniging Spierziekten Nederland (Dutch Neuro Muscular Disease Patientorganization), The NetherlandsParent and patient perspective on access to therapy Huub van Rijswiijck - VSN (NMD Patient Organization Netherlands), The NetherlandsThis session will close with a panel discussion and questions |
| 12:30 | - | 14:00 | Lunch break
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| 14:00 | - | 15:30 | Pregnancy in neuromuscular disorders
Chaired by :
Sabine Rudnik-Schöneborn – Medical University of Innsbruck, Austria
Zohar Argov – Hadassah University Hospital, Israel
PresentationsFrequent questions raised by patients before pregnancy Zohar Argov - Hadassah University Hospital, IsraelPersonal experience of pregnancy with progressive myopathy Yuriko Oda - Patient Association for Distal Myopathies (PADM), JapanPregnancy in women with muscle weakness – impossible is nothing Sabine Rudnik-Schöneborn - Medical University of Innsbruck, AustriaThis session will close with a panel discussion and questions |
| 15:30 | - | 16:00 | Coffee break
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| 16:00 | - | 17:30 | Learning from successes of clinical trials
Chaired by :
Linda Lowes – Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital and the Ohio State University, United States of America
Stojan Peric – Neurology Clinic, Clinical Centre of Serbia, Faculty of Medicine, University of Belgrade, Serbia
PresentationsLearning from CMT trials in adults and children David Cornblath - Johns Hopkins University School of Medicine, USAChallenges and limitations of re-using outcome measures and trial design Lindsay Alfano - Nationwide Children’s Hospital, USAPatient reported outcome measures in neuromuscular diseases: obstacles and perspectives Stojan Peric - Neurology Clinic, Clinical Centre of Serbia, Faculty of Medicine, University of Belgrade, SerbiaPanel discussion and questions |
| 17:30 | - | 18:00 | Close of meeting
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