Friday, 30th of May 2008

This program is permanently updated according to the answers of the solicited speakers.

Common day with the 5th International Rehabilitation Conference in Neuromuscular Diseases

Session of the day : Therapeutics

morning session : therapeutic strategies based on knowledge of molecular biology of the diseases
8h30 8h45	Opening of the 5th International Rehabilitation Conference in Neuromuscular Diseases Lecturers : Laurence Tiennot-Herment, Wahiba Boucharef
8h45 9h30	plenary lecture (auditorium) “Premature stop codon suppression for the treatment of DMD and other genetic disorders” Lecturer : H. Lee Sweeney, USA Chairpersons : Pierre Lehn and Arnold Munnich
9h30 10h15	plenary lecture (auditorium) “Antisense and RNAi technologies : from natural phenomena to new therapeutics” Lecturer : George Dickson, UK Chairpersons : Pierre Lehn and Arnold Munnich
10h15 10h45	Break
10h45 12h15	parallel symposia
	“Application of Exon skipping or reintroduction" (Auditorium) Chairperson : Anne d'Andon Judith Van Deutekom (The Netherlands) : Pre-clinical and clinical development of 2OMePS antisense oligonucleotides for Duchenne Muscular Dystrophy Luis Garcia (Frande) : Muscle function recovery in dystrophic dog after exon skipping gene therapy Daniel Schümperli (Switzerland) : Correction of SMN2 splicing by a bifunctional U7 snRNA complements a severe mouse model for SMA
	“Building tools” (Salle Endoume) Chairpersons : Kevin Flanigan et Eugenio Mercuri Christophe Béroud (France) : Knowledge bases to facilitate therapeutic approaches Serge Pauschkin (USA) : Post-transcriptional control : a new paradigm for the treatment of SMA Gideon Dreyfuss (USA) : Molecular functions of SMN and high throughput for drug discovery for SMA
	“Potential new avenues” (Amphithéâtre Callelongue) Chairpersons : Guillaume Bassez et Denis Furling Jean-Pierre Cabaniols (France) : Meganucleases for genome surgery of inherited diseases Alexandra Belayew (Belgique) : Homeodomain transcription factors DUX4, PITX1 and DUX4C : new actors in the molecular mechanism of FSHD Jack Puymirat (Canada) : Targeting of mutant DMPHK transcripts in mouse model of myotonic dystrophy type 1
12h15 13h45	lunch break

afternoon session : evaluation of the neuromuscular patient
12h15 13h45	satellite Symposium organized by PTC therapeutics (salle Morgiou)
13h45 14h30	Late breaking news Myology 2008 (auditorium) Chairpersons : Thomas Voit and Serge Braun
14h30 15h15	Plenary lecture (auditorium) “Overview of functional scales and muscle measurements” Lecturer : Eugenio Mercuri, Italy Chairpersons : Michelle Eagle et Carole Bérard
15h15 16h00	Plenary lecture Activity monitoring of neuromuscular disease” Lecturer : Diana Escolar, USA Chairpersons : Michelle Eagle et Carole Bérard
16h00 16h30	break
16h30 17h30	symposium
	“Scales for assessment of neuromuscular diseases” (Auditorium) Carole Bérard (France) : The Motor Function Measure, an outcome measure adapted to most neuromuscular diseases Richard Finkel (USA) : Scales for assessment of neuromuscular diseases : Spinal Muscular Atrophy Michelle Eagle (UK) : The North Star Ambulatory Assessment
16h30 17h30	parallel symposia
	“Evaluation of the quality of life” (Auditorium) Michael Rose (UK) : Measuring and understanding quality of life in adult muscle disease Jes Rahbek (Denmark) : Experiences with Quality of Life measurements in Denmark François Boyer (France) : Conceptual approaches of the quality of life measurements used in medicine (neuromuscular patient reported outcomes)
	“Others aspects of muscle function assessment” (Amphithéâtre Callelongue) Birgit Steffensen (Denmark) : The "Egen Klassifikation" (EK) revisited Vincent Gautheron (France) : Functional Independence Measure (MIF ™) Pierre Yves Jeannet and Anisoara Ionescu (Switzerland) : Objective quantification of daily physical activity pattern in children with Duchenne and Becker muscular dystrophies