Clinical Trials Mini-Symposium Presentations
Clinical Trials in Rett Syndrome: A Mini-Symposium
Eaglewood Resort and Spa
Itasca, IL
June 22-23, 2008
Eaglewood Resort and Spa
Itasca, IL
June 22-23, 2008
Note: (Access to presentations below is available to members only)
Goals of the Meeting
A. Percy & W. Kaufmann - Clinical Trials in Rett Syndrome: a mini-symposium.
Session I: Target Population: who should be treated?
H. Leonard - Clinical Variability and Relationships with Genotype in Rett Syndrome: insights from AussieRett and InterRett.
J. Christodoulou - Stratifying RTT Patients for Clinical Trials: a mutation based approach.
J. Neul - Clinical Trials 2008: genotype/phenotype.
A. Percy - Study Design: overview and lessons learned.
S. Naidu -
Session II: Outcome Targets: what to measure?
W. Kaufmann - Scales as Outcome Measures.
H. Leonard - Monitoring Function in Rett Syndrome for Clinical Trials.
S. Naidu -
S. Budden - Outcome Targets: what to measure?
S. Naidu -
Session III: What to Use? Prior experiences
G. Bibat -
A. Percy - Baylor/UAB Folate-Betaine Trial.
L. Villard - A randomised, multicenter, double-blind, placebo controlled, clinical trial using desipramine in girls with Rett syndrome.
Session IV: New Therapeutic Agents
M. Varney - AMPAKINE Compounds for the Treatment of Rett Syndrome.
E. Welch - PTC124: treatment of genetic disorders caused by nonsense mutations.
D. Katz -
L. Pozzo-Miller - BDNF Rescue of Dendritic Atrophy Caused by RTT-Associated MECP2 Mutations.
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