The International Foundation for CDKL5 Research is proud to announce the launch of two new CDKL5 Centers of Excellence in the Children’s Hospital of Philadelphia and the University of California Mattel Children’s Hospital in Los Angeles. The Philadelphia CDLK5 Center of Excellence was made possible due to the generous support from the Lauren Mary Francis Foundation.

Join us on Tuesday, August 6, at 8:00 pm ET for a webinar to learn more about our upcoming CDKL5 Deficiency Patient-Focused Drug Development (PFDD) meeting. On November 1, 2019, IFCR and the LouLou foundation will co-sponsor this externally-led meeting for CDD. This is a once-in-a-lifetime opportunity for caretakers to share the patient voice with a variety of stakeholders interested in developing treatments for CDD.

Karen Utley was honored to participate in the National Organization of Rare Diseases’ NORD Living Rare Forum in Houston, Texas. Several researchers stopped at the table and talked with her …

NOW OPEN!

CDKL5 Fenfluramine Study

This study will be enrolling 10 patients, ages 2-18 years old, with a confirmed genetic/clinical diagnosis of CDKL5 Deficiency Disorder (CDD) in an open label trial of fenfluramine for seizure control. Patients will be titrated over 14 days to a dose of ZX008 0.8 mg/kg/day (maximum dose 30 mg/d).

INTERNATIONAL CDKL5 DISORDER DATABASE (ICDD) AND CDKL5 DEFICIENCY DISORDER INTERNATIONAL PATIENT REGISTRY AT ODC ANNOUNCE DATA COLLECTION COLLABORATION

We are pleased to announce to the CDKL5 Deficiency Disorder (CDD) community that the International CDKL5 Disorder Database (ICDD), founded and directed by Dr. Helen Leonard and Dr. Jenny Downs in partnership with the International Foundation for CDKL5 Research (IFCR), and the CDKL5 Deficiency Disorder International Patient Registry, based at the University of Pennsylvania’s Orphan Disease Center (ODC), have outlined an agreement that will allow the two groups to collaborate on complementary projects collecting information from CDD patients.

EXCITING NEWS! YOUR RESEARCH DOLLARS AT WORK!

The National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) has awarded a five-year R01 research grant, entitled “Pathogenic Studies of CDKL5 Disorder,” to Dr. Zhaolan (Joe) Zhou, Principal Investigator (PI), at the University of Pennsylvania Perelman School of Medicine, and Dr. Douglas Coulter, co-PI, at the University of Pennsylvania Perelman School of Medicine and Children’s Hospital of Philadelphia.