09:00 – 12:00:
Physiotherapist and Investigator Training Workshop
Core dataset – adults (DM1 and DM2) and pediatrics
Topics:
10-Meter Walk/Run Test (10MWRT)
Manual Muscle Testing (11 muscle pairs)
Muscular Impairment Rating Scale (MIRS)
Handgrip strength (JAMAR dynamometer)
Sit-to-Stand Test (5 repetitions)
HOT (Hands-on Training)
North Star Ambulatory Assessment (NSAA)
13:00 – 14:00:
Lunch buffet
14:00 – 16:00:
Cognitive Training Workshop
Core dataset – adults and pediatrics
17:00 – 17:15:
Opening Session
(Valeria Sansone, Guillaume Bassez, Benedikt Schoser)
17:15 – 17:45:
Define the Meeting Objectives – Start-up Activities – Part I
Structure of the EU-CRTN
Future meetings, topics, and activities
17:45 – 19.40:
Scientific talks: Translational and Clinical Insights
in Myotonic Dystrophy
17.45-18.05: CNS: Preclinical data clinical correlates (Mario Gomes Pereira)
18.05-18.25: Muscle MRI (Matteo Garibaldi)
18.25-18.45: Brain MRI (Gabriele Siciliano)
18.45-19.05: Pediatric CNS workshop results (Valeria Sansone)
19.05-19.25: Pediatric GI workshop results – (Hilde Braakman)
19.25-19.40: Discussion – All
20:30:
Networking dinner
08:30 – 10.00:
Start-up Activities – Part II
Data governance & sharing (roles and engagement of patient organisations)
Regulatory framework (GDPR and relevant regulatory requirements,
incl. EMA guidance)
Data access & project implementation (data request procedures,
feasibility and project workflows)
Operational tools & templates (CRTN templates, SOPs, etc.)
10.00 – 11.15:
Scientific talks: Registry Updates and Clinical Research Developments
in Myotonic Dystrophies:
10.00-10.20: END-DM1 & Extension (Nicholas E. Johnson),
10.20-10.40: DM2 update and End-DM2 (Benedikt Schoser)
10.40-11.00: Registry alignment (Guillaume Bassez )
11.00-11.15: Discussion – All
11.15 – 11.30:
Coffee Break
11.30-13.10:
Scientific talks: Global Insights and Collaborative Advances in DM
11:50–12:10: Japanese Network – fostering integration (Masanori Takahashi)
12:10–12:30: DM1-Hub Project (Spain) (Gisela Nogales Gadea)
12:30–12:50: Drug Development Update: New Treatments (Karlien Mul)
12:50–13:00: Discussion
13.00-14.00
Working Lunch
14.00-16.00
Industry Perspective Session
14:00 – 15:00: Site Readiness – Bridging the Gap for Clinical Site Preparedness
15:00 – 16:00: Round table: Ask the Industry – Gaining Insights and Perspectives
16:00 – 17:00:
Wrap-up & Next Steps
