Thursday 31st October

Session two
Patient involvement and ethics in TREAT-NMD related research

Chaired by Anna Ambrosini
Fondazione Telethon, Italy
08.40 - 09.00 Patient Forum
Filippo Buccella – Parent Project Onlus, Italy
09.00 - 09.20 Advocacy, expectations and hype: improving the communication of clinical results in NMDs
Pauline McCormack – Newcastle University, UK
09.20 - 09.40 Podium discussion

Session three
Upcoming new themes of collaborative research

Chaired by Ichizo Nishino
National Institute of Neurscience, National Center for Neurology and Psychiatry, Japan
09.40 - 10.00 Next-generation sequencing and Neuromics
Nigel Laing - University of Western Australia, Australia

10.00 - 11.00
Coffee break
11.00 - 11.20 Emerging therapies in SMA
Francesco Muntoni – Institute of Child Health, University College London, UK

11.20 - 11.40 Emerging therapies in Myotonic Dystrophy
Guillame Bassez – Université Paris-Est Créteil Val de Marne, France
11.40 - 12.00 Diagnostic approaches in Pompe disease
Teresinha Evangelista – Newcastle University, UK

12.00 - 12.20 Gene and cell therapies for muscular dystrophy
Thomas Voit – Institute de Myology, France
12.20 - 13.00 Podium discussion
13.00 - 14.30
Lunch
Chaired by Kevin Flanigan
Ohio State University, USA
14.30 - 14.50 MRI as an outcome measure in clinical trials (including natural history studies in LGMD2B, LGMD2I)
Volker Straub – Newcastle University, UK

14.50 - 15.10 Trial readiness in FSHD
Rabi Tawil – University of Rochester Medical Center, Rochester, NY, USA

15.10 - 15.30 CINRG and international trial networks for NMD
Eric Hoffman- Children's National Medical Center, Washington DC, USA

15.30 - 15.50 Utility of patient registries for clinical care and post-marketing surveillance
Jan Verschuuren – Leiden University Medical Centre, Netherlands

15.50 - 16.30 Podium discussion
16.30 - 17.00
Coffee break

17.00 - 19.30

TREAT-NMD Global Database Oversight Committee (TGDOC) meeting (by invitation only)
Chaired by: Jan Verschuuren (Leiden University Medical Centre, Netherlands) and Hugh Dawkins (Office of Population Health Genomics, Department of Health Western Australia, Australia)
19.45
Conference dinner - Great North Museum
 
12 Apr 2017