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09:30 - 09:50
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Overview and short history of repeat expansion diseases - Jean-Louis Mandel, IGBMC |
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09:50 - 10:25
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Mechanisms of repeat instability - Christopher Pearson, Hospital for Sick Children/Canada |
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10:25 - 11:00
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SBMA (spinobulbar muscular atrophy): from gene discovery to mechanism and treatment - Kurt Fischbeck, NIH/USA |
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11:00 - 11:15
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Pause café |
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11:15 - 11:50
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Animal models for Fragile X syndrome and FXTAS: What have they taught us, current and future developments. - Ben Oostra, Erasmus University/The Netherlands |
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11:50 - 12:25
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20 years of anticipation: from mutation to therapy - Keith Johnson, Novartis/USA |
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12:25 - 13:00
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Friedreich ataxia: from GAA repeats to pathomechanisms and therapeutic prospects - Michel Koenig & Helene Puccio, IGBMC |
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13:00 - 14:00
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Déjeuner |
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14:00 - 14:35
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Overview of current clinical trials in Europe for HD, polyglutamine SCAs (spinocerebellar ataxias) and Friedreich ataxia - Alexandra Durr, Hopital Pitié Salpétrière/ICM/France |
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14:35 - 15:10
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Unexpected mechanisms (antisense transcripts, RAN translation) - Laura Ranum, University of Florida/USA |
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15:10 - 15:45
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Toxic mRNAs mechanisms in myotonic dystrophies and in Fragile X tremor ataxia syndrome (FXTAS) - Nicolas Charlet, IGBMC |
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15:45 - 15:55
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Pause café |
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15:55 - 16:20
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Use of human embryonic stem cells for development of novel therapeutic strategies in Myotonic Dystrophy Type 1 and in Huntington disease. - Cécile Martinat, I-STEM/France |
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16:20 - 16:40
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Perspectives of treatment with antisense oligos in Myotonic Dystrophy. - Denis Furling, Institut de Myologie/France |
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