Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe) and Metabolic Profiling of Rett syndrome, MECP2 duplication syndrome, and Rett-related disorders.

$106,106U54FY2016HDNIH

University Of Alabama At Birmingham, Birmingham AL

Investigators

Linked publications & trials

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Abstract

An important component of the Rare Disease Clinical Research Consortium for Rare Diseases Clinical Research Network is pilot projects. The pilot projects within this proposal will be administered through the IRSF subcontract. IRSF has a capped indirect rate of 10%, so this will be applied to these projects. Two pilot studies are proposed in this application, each spanning 2 years. Additional money is budgeted for the final 2 years of this funding period for additional pilot studies to be chosen during year 3. We have opted to leave these funds initially uncommitted in order to maintain flexibility to fund exciting ideas that may arise during this funding period, however, there are some potential ideas, which are listed below.

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