2022 was another robust year for CDKL5 research! Over the course of the year, progress was made in model development, biomarker identification, further understanding of the underlying mechanisms of CDD, and identification of potential therapeutic targets.

In addition, the International CDKL5 Clinical Research Network (ICCRN) validated a new outcome measure (CDD-Hand), published on quality of life, and collaborated with international partners on a landmark publication about consensus recommendations for the management of CDD.

Our Science Director, Heidi Grabenstatter, has summarized many of the year's publications for our community. See it here: CDKL5 Publications in 2022

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Patient advocacy is at the heart of our mission for CDKL5. We know that it is critical for patient advocacy groups to have a seat at the table when discussions are happening about their disease. The IFCR is active with key stakeholders in our work for CDKL5.

At the end of last year, we attended the FDA's Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) Patient-Focused Drug Development listening meeting - Patient Perspectives on Gene Therapy Products. We submitted written comments to support clinical trial design for disease-modifying treatments.

Click to read our submitted comments.