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 1.Follow up of 2006 San Francisco meeting was needed for discussing multi-center initiatives and the future of RettSearch. 2.The Rett community is growing: new clinics and more scientists (opportunities for translational research). 3.Merge of IRSA and RSRF into IRSF provided opportunity. IRSF is committed to patient-based research. 4.Mini-symposium format allows the identification of key issues. Next meeting will be expanded. 5.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: a)Carrying out multi-center RTT clinical trials. b)Introducing new therapies for RTT. 2.To develop a document stating the meeting’s: a)Conclusions (diagnosis). b)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? a)Prior experiences. b)New and promising therapeutic agents.