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Research dedicated to furthering the understanding of CDKL5 function to inform the development of targeted, novel therapies.
Transformative research to enhance our understanding of the cellular, molecular, genetic and systems-level mechanisms contributing to the pathogenesis of CDKL5 Deficiency Disorder (CDD), facilitating the continued investigation of disease-modifying strategies.
Research aimed at improving CDD disease models (e.g., cell-based, tissue-based, or animal models) in an effort to assess the potential efficacy of therapeutic interventions against phenotypic deficits in the CDD patient population.