Watch the U01 Study Update!

In 2019, the IFCR funded a grant to build and foster a collaborative network between our CDKL5 Centers of Excellence and the International CDKL5 Disorder Database in Australia. This network became the International CDKL5 Clinical Research Network (ICCRN). With disease-modifying treatments like gene therapies on the horizon, it was critical to ensure our community was prepared and clinical trial-ready. This was not a small lift. For this to be as successful as possible, we knew our community would have to show up for visits, record and submit motor videos, and answer multiple surveys to help develop and validate clinical outcome measures, as well as establish the beginnings of a natural history for the diagnosis.

Thankfully, our community came through! The success of the initial phase of the project made it possible for the ICCRN to receive a grant from the National Institute of Health (“U01 grant”) to help support the project. This became the “One of 200” campaign, which many of you have participated in. You all are the reason this has been successful so far. As we enter our final year of funding, we want the community to know how very grateful we are! This video provides a short update from Dr. Benke with a sincere expression of gratitude and encouragement to maintain momentum through the completion of this grant.