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CDKL5 Voices | August 2023

Congratulations Dr. Helen Leonard!

Join us in offering hearty congratulations to our own Dr. Helen Leonard for being awarded the 2023 Rare Champion of Hope in Research award by Global Genes.

"This award recognizes trailblazers and leaders in rare disease research, drug development and scientific processes who have taken an innovative approach to advance outcomes for the rare disease community." -globalgenes.org

If your child has CDD, chances are you've had a phone call from Dr. Leonard or her team in Australia! If not, please enroll in the International CDKL5 Disorder Database (ICDD)! The ICDD has utilized data provided by caregivers to inform multiple clinical trials and key publications in the scientific and medical literature. We need your participation!

Dr. Leonard also collaborates as part of the International CDKL5 Clinical Research Network (ICCRN) to develop and validate CDKL5-specific clinical outcome measures that we hope will be used in future clinical trials, including gene therapy trials.

Congratulations on this honor, Dr. Leonard! Well deserved! Thank you for all you do for our CDKL5 community!

Awardee Details

Back-to-School - Is Your Seizure Action Plan Up-to-Date?

It's back-to-school time and for students with CDKL5 deficiency disorder, it is a time to ensure all medical orders, including a Seizure Action Plan, are up-to-date.

"A Seizure Action Plan (SAP) contains tailored guidelines on how to respond during a seizure, based on the patient's medical history. It includes health and medical information specific to the patient and helps others recognize seizures and the appropriate steps to take to keep him or her safe from injury or damage caused by prolonged seizures." -seizureactionplans.org

You can find samples of Seizure Action Plans HERE.

European Commission Approves Ztalmy

The European Commission has granted approval of Ztalmy® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age. ZTALMY may be continued in patients 18 years of age and older.

“The approval by the European Commission represents a significant milestone for children, families and physicians who, until now, have long been challenged by the impact of seizures and lack of treatments available for CDD,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “This achievement reflects our organization’s unwavering commitment to bring an effective treatment option to individuals living with CDD and we are grateful to all the stakeholders who made the approval possible.”

In July 2021, Marinus and Orion Corporation entered into a collaboration agreement which grants Orion the right to commercialize ZTALMY in Europe.

Learn More

New on the IFCR Blog

CDKL5 Publication Breakdown: specific inhibitor of CDKL5

In this blog series, IFCR Science Director, Heidi Grabenstatter, breaks down CDKL5 publications in easy-to-understand language. In this blog, she summarizes Discovery and characterization of a specific inhibitor of serine-threonine kinase cyclin dependent kinase-like 5 (CDKL5) demonstrates role in hippocampal CA1 physiology. Read it HERE.

Family Support Program

We know how hard life with CDKL5 can be and we are here for you whenever you need us.

Would you like to speak with a fellow CDKL5 parent who knows what you're facing? We have peer support volunteers who are available to offer a listening ear or resources that can help. Email FSR@CDKL5.com.

FOR FAMILIES

Enrolling CDKL5 Clinical Trials

Did you know? Our website is a great place to learn what clinical trials are enrolling for CDKL5!

There are opportunities for interventional trials as well as clinical research, including ways to participate in CDKL5 research from your home, anywhere in the world!

This information and even more can be found on our website. Check back frequently for updates as new opportunities emerge!

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Have you joined "Connect CDKL5"?
Connect families receive direct communication about clinical trial opportunities for CDKL5 and other important information important for families. Join today!

PARENTS, JOIN CONNECT CDKL5

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