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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| Hector, Ralph D; Kalscheuer, Vera M; Hennig, Friederike et al. (2017) CDKL5 variants: Improving our understanding of a rare neurologic disorder. Neurol Genet 3:e200 |
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