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Friday, May 30 >>> morning<br />
Common day with the 5 th International Rehabilitation Conference in Neuromuscular<br />
Therapeutic strategies based on knowledge<br />
of molecular biology of the diseases<br />
8.30 am-8.45 am Opening of the 5 th International Rehabilitation<br />
Conference in Neuromuscular Diseases<br />
8.45 am-10.15 am Plenary lectures XAuditorium<br />
Chairpersons > Pierre Lehn and Arnold Munnich<br />
8.45 am-9.30 am H. Lee Sweeney, USA<br />
> Premature stop codon suppression for the treatment of DMD and other genetic disor<strong>de</strong>rs<br />
9.30 am-10.15 am George Dickson, UK<br />
> Antisense and RNAi technologies: from natural phenomena to new therapeutics<br />
10.15 am-10.45 am Break<br />
10.45 am-12.15 am Parallel Symposia<br />
‹Applications of exon skipping or reintroduction XAuditorium<br />
Chairpersons > Anne d’Andon and Giulio Cossu<br />
Judith van Deutekom, The Netherlands<br />
> Pre-clinical and clinical <strong>de</strong>velopment of 2OMePS antisense oligonucleoti<strong>de</strong>s for DMD<br />
Luis Garcia, France<br />
> Muscle function recovery in dystrophic dog after exon skipping gene therapy<br />
Daniel Schümperli, Switzerland<br />
> Correction of SMN2 splicing by a bifunctional U7 snRNA complements<br />
a severe mouse mo<strong>de</strong>l for SMA<br />
‹Building tools XSalle Endoume<br />
Chairpersons > Kevin Flanigan and Eugenio Mercuri<br />
Programme > Friday, May 30<br />
12.15 am-1.00 pm Lunch break<br />
Christophe Béroud, France<br />
> Knowledge bases to facilitate therapeutic approaches<br />
Sergey Pauschkin, USA<br />
> Post-transcriptional control: a new paradigm for the treatment of SMA<br />
Gi<strong>de</strong>on Dreyfuss, USA<br />
> Molecular functions of SMN and high throughput for drug discovery for SMA<br />
‹Potential new avenues XAmphitheatre Callelongue<br />
Chairpersons > Guillaume Bassez and Denis Furling<br />
Jean-Pierre Cabaniols, France<br />
> Meganucleases for genome <strong>sur</strong>gery of inherited diseases<br />
Alexandra Belayew, Belgium<br />
> Homeodomain transcription factors DUX4, PITX1 and DUX4C:<br />
new actors in the molecular mechanism of FSHD<br />
Jack Puymirat, Canada<br />
> Targeting of mutant DMPHK transcripts in mouse mo<strong>de</strong>l of myotonic dystrophy type 1<br />
12.15 pm - 1.45 pm Satellite Symposium organized by PTC Therapeutics XSalle Morgiou<br />
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