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CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008

CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008. Organized by: RettSearch Sponsored by: International Rett Syndrome Foundation. CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008. CONTEXT.

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CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008

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  1. CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008 Organized by: RettSearch Sponsored by: International Rett Syndrome Foundation

  2. CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008 CONTEXT Follow up of 2006 San Francisco meeting was needed for discussing multi-center initiatives and the future of RettSearch. The Rett community is growing: new clinics and more scientists (opportunities for translational research). Merge of IRSA and RSRF into IRSF provided opportunity. IRSF is committed to patient-based research. Mini-symposium format allows the identification of key issues. Next meeting will be expanded. Potential industry partners and new clinic centers are present.

  3. CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008 GOALS 1. To determine where we are in terms of: • Carrying out multi-center RTT clinical trials. • Introducing new therapies for RTT. 2. To develop a document stating the meeting’s: • Conclusions (diagnosis). • Recommendations (treatment). 3. To publish Conclusions & Recommendations of meeting and full-length? papers (J Child Neurol).

  4. CLINICAL TRIALS IN RETT SYNDROME A Mini-Symposium Chicago, June 22-23, 2008 AGENDA 1. Target population: who should be treated?a) Genotype-phenotype correlations b) Natural history studies 2. Outcome targets: what to measure?a) Rating scales b) Other measures 3. Treatment targets: what to use? • Prior experiences. • New and promising therapeutic agents.

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