Our 2019 programme is currently being developed. Below are programme details of our previous conference to give you an idea of what might be involved.
Please note that our 2019 programme may well differ from this in places.
You can still download a PDF of the programme here.
Monday 27th November 2017
| 13:00 | - | 13:15 | Conference opening
Chaired by :
Janbernd Kirschner – University Medical Center Freiburg, Germany
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| 13:15 | - | 15:00 | Ten years of TREAT-NMD – Looking back – looking forward
Chaired by :
Janbernd Kirschner – University Medical Center Freiburg, Germany
Alain Geille – AFM Téléthon, France
Presentations10 Years of TREAT-NMD Volker Straub - John Walton Muscular Dystrophy Research Centre, Newcastle University, United Kingdom Patients in neuromuscular diseases: an army of experts Alain Geille - AFM Téléthon, France High quality evidence and strong recommendations in rare diseases: mission impossible?
Jorg Meerpohl - Cochrane Center, Germany |
| 15:00 | - | 15:30 | Coffee break
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| 15:30 | - | 17:30 | Emerging treatment strategies for NMD treatment
Chaired by :
Markus Rüegg – University of Basel, Switzerland
John Porter – Myotonic Dystrophy Foundation, United States
PresentationsLAMA2 MD: Understanding mechanism as a guide to treatment Markus Rüegg - University of Basel, Switzerland Navigating the path towards novel therapies for NMDs John Porter - Myotonic Dystrophy Foundation, United States Gene therapy of X-linked myotubular myopathy : from preclinical studies in animal models to a clinical trial Ana Buj Bello - , Myotonic dystrophy: from basic mechanisms to treatment options
Charles Thornton - University of Rochester, USA |
| 18:00 | - | 20:00 | Networking reception
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Tuesday 28th November 2017
| 8:30 | - | 10:30 | Non-drug treatments and innovative outcome measures
Chaired by :
Baziel van Engelen – Radboud University Nijmegen Medical Centre, Netherlands
Eugenio Mercuri – Università Cattolica del Sacro Cuore, Italy
PresentationsOptimistic Experience in DM1 Baziel van Engelen - Radboud University Nijmegen Medical Centre, Netherlands Outcome measures Eugenio Mercuri - Università Cattolica del Sacro Cuore, Italy PROMS new methods Nathalie Goemans - University Hospitals Leuven, Belgium Fatigue, how to measure? How to treat? Hans Knoop - Academic Medical Center Amsterdam, Netherlands Exercise in various diseases
John Vissing - University of Copenhagen, Denmark |
| 10:30 | - | 11:00 | Coffee break
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| 11:00 | - | 12:30 | Regulatory exchange matters
Chaired by :
Annemieke Aartsma-Rus – Leiden University Medical Center, Netherlands
Pavel Balabanov – European Medicines Agency, UK
PresentationsPatient involvement in therapy development and the regulatory process Elizabeth Vroom - United Parent Projects Muscular Dystrophy, Netherlands The development of nusinersen for SMA
Eugenio Mercuri - Università Cattolica del Sacro Cuore, Italy |
| 12:30 | - | 14:00 | Lunch break
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| 14:00 | - | 15:30 | Looking for a new deal for better access to rare diseases innovative therapies?
Chaired by :
Simone Boselli – EURORDIS, France
PresentationsValue assessment in Europe – common principles for orphan drug assessment Michael Schlandler - University of Heidelberg, Germany Early dialogue between payers and manufactures: the MoCA experience Anna Bucsics - University of Vienna, Breaking the Access Deadlock: patients recommendations to leave no one behind
Simone Boselli - EURORDIS, France |
| 15:30 | - | 16:00 | Coffee break
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| 16:00 | - | 18:00 | Data Integration and Gene Discovery
Chaired by :
Hanns Lochmüller – John Walton Muscular Dystrophy Research Centre, Newcastle University, UK
Carsten Bonnemann – National Institute of Neurological Disorders & Stroke - NINDS, United States
PresentationsGene identification in inherited neuropathies & complex neuromuscular syndromes: from linkage to exomes & genomics Jan Senderek - Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik, Germany Combining RNAseq & exome/genome sequencing congenital myopathies Carsten Bonnemann - National Institute of Neurological Disorders & Stroke - NINDS, United States Data sharing & integration, exomes, deep phenotypes & RD-Connect, congenital myasthenic syndromes Hanns Lochmüller - John Walton Muscular Dystrophy Research Centre, Newcastle University, UK Multi-omics integration & systems medicine, mitochondrial myopathies Rita Horvath - John Walton Muscular Dystrophy Research Centre, Newcastle University, UK Panel
Christophe Béroud - , |
| 19:30 | - | 0:30 | Conference Dinner
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Wednesday 29th November 2017
| 8:30 | - | 10:30 | Biomarkers – Animal models
Chaired by :
Kevin Flanigan – Ohio State University College of Medicine, United States
Alessandra Ferlini – University of Ferrara, Italy
PresentationsInteraction between academia and industry in biomarker research for DMD Pietro Spitali - Leiden Univeristy Medical Centre, Netherlands Identifying common molecular determinants of axonal CMT Manisha Juneja - , Pharmacodynamic biomarkers in the vamorolone program: Multiple contexts of use Eric Hoffman - Binghamton University - SUNY; ReveraGen BioPharma, United States Biomarkers in Spinal Muscular Atrophy: From Bedside-to-Bench-to-Bedside
David Arnold - The Ohio State University, USA |
| 10:30 | - | 11:00 | Coffee Break
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| 11:00 | - | 13:00 | Standards of Care
Chaired by :
Thomas Sejersen – Karolinska Institute, Sweden
PresentationsCare guidelines for NMDs – the Present landscape and the way forward Thomas Sejersen - Karolinska Institute, Sweden How can EURO-NMD help develop and implement care guidelines for NMDs? Teresinha Evangelista - John Walton Muscular Dystrophy Research Centre, Newcastle University, UK Principles and practical aids on developing care guidelines for NMDs
Karen Ritchie - Healthcare Improvement Scotland, UK |
| 13:00 | - | 14:30 | Lunch break
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| 14:30 | - | 17:00 | Product specific presentations
Chaired by :
Rudolf Korinthenberg – University Hospital Freiburg, Germany
Nathalie Goemans – University Hospitals Leuven, Belgium
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