Our 2019 programme has now been finalised and can be found below.
The PDF version is available here.
Venue
Leiden Stadsgehoorzaal
Breestraat 60
2311 CS Leiden
The Netherlands
Monday 9th December 2019
| 17:00 | - | 19:00 | Early Registration in the Entreehal Breestraat |
| 18:00 | - | 19:30 | Drinks Reception in the Catharina Foyer |
Tuesday 10th December 2019
| 8:00 | - | 9:00 | Registration in the Entreehal Breestraat |
| 9:00 | - | 9:15 | Welcome address
Chaired by :
Annemieke Aartsma-Rus – Leiden University Medical Center, Netherlands
|
| 9:15 | - | 10:15 | Keynote Lecture
PresentationsWhen being just a scientist is not enough
Mayana Zatz – Professor of Genetics, University of São Paulo, Brazil
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| 10:15 | - | 10:45 | Coffee break and posters in the Catharina Foyer |
| 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
PresentationsPatient engagement in medicine development
Ingrid de Groot – Patient representative, The Netherlands
Kees van der Graaf – Chairman of FSHD Unlimited, The Netherlands This session with close with a panel discussion and questions. |
| 12:15 | - | 14:00 | Lunch break Lunch will be available in Conelis Schuytzaal. In addition to lunch being served the following presentations will form part of today’s lunchtime symposium… PresentationsSarepta -Evolution of Registries in Neuromuscular Disorders
Michela Guglieri – John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom
Biogen’s vision to transform the therapeutic landscape of neuromuscular diseases
Wildon Farwell – Late Stage Unit Head for Pain, Neuromuscular & Rare Disease, Biogen,
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| 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
PresentationsTake a deep breath and cough! Inspiratory muscle training and cough assistance in NMD
Anri Human – Sefako Makgatho Health Sciences University, South Africa
Nutritional assessment and interventions in adult NMD
Heidi Zweers – Radboud University Nijmegen Medical Centre, Netherlands
Exercise in neuromuscular disease: a moving topic
Nicole Voet – Radboud University Medical Center, Netherlands
This session with close with a panel discussion and questions. |
| 15:30 | - | 16:00 | Coffee break and posters in the Catharina Foyer |
| 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, Spain
Supporting the collection of post-marketing surveillance data
Craig Campbell – M.D. Head Division of Pediatric Neurology and TGDOC Chair, Western University, Canada
The EURO-NMD Clinical Patient Management System (CPMS)
Teresinha Evangelista – Researcher and EURO-NMD Coordinator, INSERM Institute of myology, France
This session will close with a panel discussion and questions |
| 19:30 | - | 23:00 | Networking dinner in the Breezaal room |
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
PresentationsPatient-customized oligonucleotide therapy for rare genetic diseases
Jinkuk Kim – Research Fellow, Boston Children’s Hospital and Harvard Medical School, United States of America
Opportunities and challenges for personalised medicine
Annemieke Aartsma-Rus – Leiden University Medical Center, Netherlands
CRISPR-Based Genome Editing for Duchenne Muscular Dystrophy – Successes
Charles Gersbach – Duke University, Durham NC, United States
This session will close with a panel discussion and questions |
| 10:30 | - | 11:00 | Coffee break and Posters in the Catharina Foyer |
| 11:00 | - | 12:30 | Access to drug treatment
Chaired by :
Ans van der Ploeg – Erasmus MC Univeristy Medical Center, Rotterdam, 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, France
Clinicians’ perspective on access to therapies
Ans van der Ploeg – Erasmus MC Univeristy Medical Center, Rotterdam, Netherlands
Parent and patient perspective on access to therapy
Huub van Rijswiijck – VSN (NMD Patient Organization Netherlands), The Netherlands
This session will close with a panel discussion and questions |
| 12:30 | - | 14:00 | Lunch break Lunch will be available in Conelis Schuytzaal. In addition to lunch being served the following presentations will form part of today’s lunchtime symposium… PresentationsSanthera – DMD Beyond the wheelchair
Erik Niks – Leiden University Medical Center, Netherlands
PTC Therapeutics – A science led, patient centric approach to treating rare diseases
Christian Werner – PTC Therapeutics, Germany
Roche – How do we set the right expectations when treating SMA
Nathalie Goemans – University Hospitals Leuven, Belgium
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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, Israel
Personal experience of pregnancy with progressive myopathy
Yuriko Oda – Patient Association for Distal Myopathies (PADM), Japan
Pregnancy in women with muscle weakness – impossible is nothing
Sabine Rudnik-Schöneborn – Medical University of Innsbruck, Austria
This session will close with a panel discussion and questions |
| 15:30 | - | 16:00 | Coffee break and posters in the Catharina Foyer |
| 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, USA
Challenges and limitations of re-using outcome measures and trial design
Lindsay Alfano – Nationwide Children’s Hospital, USA
Patient reported outcome measures in neuromuscular diseases: obstacles and perspectives
Stojan Peric – Neurology Clinic, Clinical Centre of Serbia, Faculty of Medicine, University of Belgrade, Serbia
Panel discussion and questions |
| 17:30 | - | 17:45 | Close of meeting Professor Volker Straub will close our conference |
