222ndENMC International Workshop:
Myotonic Dystrophy, developing an European consortium
for care and therapy
1 – 3 July 2016
*programme *
Friday 1 July 2016
12:30 - 14:00Lunch
14:00 - 15:00 Introductory Session
14:00 - 14:10 Welcome by dr. Alexandra Breukel, Managing Director ENMC
14:10 - 14:15Welcome Organisers
14:15 - 14:25Patient perspectives, Jeanette Charlton (UK)
14:25 - 14:35Patient perspectives, Alain Geille (Fr)
14.35 - 14:45Introduction to the Wyck foundation, John Porter (USA)
14:45 - 15:15Why establish a network for DM trials, examples from other conditions, Benedikt Schoser
Benedikt Schoser
15:15 - 19:00 The last five years, trial readiness, outcome measures and patient registries
15:15 - 15:30Imaging: Muscle MRI, Arend Heerschap
15:30 - 15:45Imaging: Brain MRI, Cornelia Kornblum
15:45 - 16:00Imaging: Skeletal muscle MRI, Arend Heerschap
16:00 - 16:30Coffee/Teabreak
16:30 - 16:45Outcome measures: Respiratory, Valeria Sansone
16:45 - 17:00Outcome measures: Cognitive outcomes, Giovanni Meola
17:00 - 17:15Outcome measures: Myotonia, Christopher Lindberg/David Hilton-Jones
17:15 - 17:30 Outcome measures: Muscle Strength and Function, Marie Kierkegaard
17:30 - 17:45 Outcome measures : Activity and cognitive outcomes, Baziel van Engelen
17:45 - 18:00Outcome measures: Biomarker and genetics, Darren Monckton
18.00 - 18.15Outcome measure: Sleep, Matthias Boentert/Antonio Atalaia
18.15 - 18.30Outcome measures: Lessons from OMMYD, Cynthia Gagnon
18.30 – 18.45Value of patient reported outcomes, Chad Heatwole
18:45 – 19.00Classification in DM1, a model for cohort stratification. How to stratify cohorts, Guillaume Bassez
19:00 – 19:30Discussion: OM selection, do we need a minimal dataset, characterisation and stratification Chari: Guillaume Bassez
20:00 Dinner
Saturday 2 July 2016
09.00 - 11.00Registries (cont. from Friday)
09:00 - 09:15Registries: Swedish national registry, Christopher Lindberg
09:15 - 09:30Registries: Serbian national registry, ReaDy, Vidosava Stojanović
09.30 - 09:45Registries: Danish national registry/cohort, John Vissing
09:45 - 10.00Registries: French national registry/cohort, Guillaume Bassez
10:00 - 10:15 Registries: A global review of DM registries “Naarden five years later”, Libby Wood Libby Wood
10:15 - 11:00Registries Discussion: the next steps to create a global registry, Hanns Lochmüller
11:00 - 11:30 Coffee/Teabreak
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11:30 - 12:00 What has been learnt from OPTIMISTIC; trial design, set-up, recruitment, retention, Baziel van Engelen/Shaun Treweek
12:00 - 12:30Discussion: How to make things better next time, what else need to be considered for sponsor led trials, all
12:30 - 13:00Discussion: how do we stratify patients into suitable cohorts, power calculations, what do we learn from unpublished data, what trial designs would be suitable, all
13:00 - 14:00 Lunch
14:00 - 15:00 Beyond the trails: what do we need to consider
14:00 - 14:30 Regulatory Interactions and post marketing surveillance, Pavel Balabanov
14:30 - 15:00 Discussion: Where do we start? Chair: Hanns Lochmüller
15:00 - 15:30 Coffee
15:30 - 17:00What has already been developed, what can we re-use?
15:30 - 16:00What are other European Networks Offering? What is the benefit of sharing data and samples existing structures e.g. RD-Connect, Rachel Thompson
16:00 - 16:30 What is required to share samples? Hanns Lochmüller
16:30 - 17:00 Discussion: How can the DM community use these infrastructures and tools? Chair: Baziel van Engelen and Benedikt Schoser
17:00 - 18:00Current and expected trials, an industry perspective
17:00 - 17:30ISIS/Biogen, Laury Mignon
17:30 - 18:00Discussion: What industry need to be trial ready, Chair: Benedikt Schoser
19.00Dinner
Sunday 3 July 2016
09:00 - 10:30Establish an European DM Consortium
Discussion: Structure and Governance
Chair: Benedikt Schoser, Baziel van Engelen
10:30 - 11:00Coffee
11:00 - 12:00Work plan
11:00 - 11:20Lay Summary and final patients comments, Alan Geille/Jeanette Charlton
11:20 - 11:40Full Workshop publication, Organisers
11:40 - 12:00General Discussion, Organisers
12:00 - 13:00End of meeting, lunch and departure of participants