Wednesday 30th October

09:00 - 11.30 CARE-NMD Project Meeting (by invitation only)
Chaired by: Janbernd Kirschner - University Medical Centre Freiburg, Germany
09.00 - 12.00
Meeting Registration
12.00 - 13.00
Lunch

Session one:
Scientific results and achievements of TREAT-NMD related research

Chaired by Eric Hoffman
Children's National Medical Center, USA
13.00 - 13.10 Opening remarks
Hanns Lochmüller - Newcastle University, UK
13.10 - 13.30 CARE-NMD results
Janbernd Kirschner - University Medical Centre Freiburg, Germany
13.30 - 13.50 Burden of Illness in DMD
Kate Bushby - Newcastle University, UK
13.50 - 14.10 What did we learn from the DMD registries?
Hanns Lochmüller - Newcastle University, UK
14.10 - 14.40 Podium discussion:
DMD and trial infrastructure
Chaired by Annemieke Aartsma-Rus
Leiden University Medical Centre, Netherlands
14.40 - 15.00 Impact of TACT
Dominic Wells - Royal Veterinary College, University of London, UK
15.00 - 15.20 AON clinical trials in DMD
Nathalie Goemans - University Hospitals, Leuven, Belgium
15.20 - 16.20
Coffee break
16.20 - 16.40 COST and EMA regulatory interaction
Annemieke Aartsma-Rus - Leiden University Medical Center, The Netherlands
16.40 - 17.00 Standardization of animal models for translation
Markus Ruegg - Biozentrum, University of Basel, Switzerland
17.00 - 17.20 Natural history studies and therapy development on GNE myopathy
Ichizo Nishino - National Institute of Neuroscience, National Center for Neurology and Psychiatry, Japan
17.20 - 17.40 New outcome measures for clinical trials in SMA and DMD
Eugenio Mercuri - Università Cattyolica del Sacro Cuore, Italy
17.40 - 18.20 Podium discussion:
Preclinical and clinical tools
18.20 - 20.00
Drinks reception
 
12 Apr 2017