TREAT-NMD 7th International Conference 2022 programme.
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
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| 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 |
