Investigators: Helen Leonard, Jacinta Saldaris, Jenny Downs, Peter Jacoby
Project description
There are increasing efforts worldwide to develop new treatments for CDKL5 Deficiency Disorder (CDD), to reduce symptoms and possibly even reverse symptoms. There is a critical need to ensure that these new treatments can be tested properly in clinical trials that use relevant outcome measures that have been tested specifically for CDD. This project will be a stepping-stone to enable readiness for CDD clinical trials, by validating outcome measures for CDD. We are now planning studies to generate, validate and refine clinical outcome measures necessary to comprehensively assess changes in response to newly developed treatments for CDD.
External collaborators
- Dr Tim Benke (University of Colorado, Children's Hospital Colorado)
- Dr Scott Demarest (University of Colorado, Children's Hospital Colorado)