We are pleased to announce that the Orphan Disease Center and Loulou Foundation has released a call for grant that will support research related to CDKL5 Deficiency Disorder (CDD). CDKL5 Deficiency Disorder (CDD) is a monogenic, neurodevelopmental disorder characterized by treatment-resistant epilepsy and severe neurodevelopmental delay. The disease is driven by the loss of a kinase called CDKL5 which is responsible for normal neuronal development, synapse formation and signal transmission. The mechanisms by which loss of CDKL5 expression leads to this CNS disorder remain unclear. The gene encoding this protein is located on the X chromosome, with heterozygous females primarily affected. The disease does not exhibit neurodegeneration, and animal models strongly suggest the potential for reversibility. There are no approved therapies and the current standard of care is not effective at managing seizures or improving neurodevelopmental or motor deficits.
Letters of Interest (LOIs) are due no later than FRIDAY, FEBRUARY 26, 2021, by 5pm EST. Grant criteria, the entire RFA, and additional program details can be found here.
For more details you may download the attached file: