NIH Awards Clinical Trial Readiness UO1 Grant to the International CDKL5 Clinical Research Network

“This NIH Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical studies that address critical needs for clinical trial readiness in rare neurological and neuromuscular diseases. These studies should result in clinically validated biomarkers and clinical outcome assessment measures appropriate for use in upcoming clinical trials. Through the support of trial readiness studies, NINDS and NCATS expect to enhance the quality and increase the likelihood of success of clinical trials in these rare diseases.”

The Co-Directors of the International CDKL5 Disorder Database (ICDD)- a rich natural history database collecting parent-reported and genetic data and the Directors of the eight IFCR-funded CDKL5 Centers of Excellence (COEs) – a network of clinics throughout the USA that specialize in the care of patients with CDKL5 Deficiency Disorder (CDD) actively participate in a collaborative research infrastructure, the International CDKL5 Clinical Research Network (ICCRN). This network was initiated through IFCR funding. The ICCRN seeks to answer clinically significant questions to drive improvements in the clinical care of patients with CDD. To date, IFCR support has led to 12 publications since 2017 fostered by this collaboration. Disease-modifying therapies are currently in development for CDD patients, but a mission-critical step remains unfilled prior to the FDA-approved initiation of clinical trials.

There are no measures validated for CDD and the severity of this condition renders those outcome measures that exist unsuitable.

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CDKL5 Deficiency Disorder Voice of the Patient Report Submitted to FDA

Understanding the patient voice is fundamental for therapy development. Companies need to ensure that what matters most to patients is being assessed, and regulators and payers asks for demonstration of value to support approval and reimbursement. Today, the Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) are proud to release the “CDKL5 Deficiency Disorder Voice of the Patient Report”, capturing the voice of patients living with this rare genetic disease that affects thousands of people.

The report is the outcome of a groundbreaking patient-centered meeting …

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IFCR & RDMD Partner to Accelerate Drug Development

You can accelerate drug development in CDKL5 (CDKL5 deficiency disorder)

Important research records are trapped in hospitals across the country. As a result, drug researchers can’t access the information they need to advance treatments.

But as a patient caregiver, you can. Unlock your records and contribute them to research, privately and securely.

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Marinus Announces Completion of Marigold Trial

Marinus Pharmaceuticals Completes Targeted Enrollment in Pivotal Phase 3 Study for CDKL5 Deficiency Disorder

“Enrolling 100 patients in our registrational, pivotal Phase 3 trial evaluating ganaxolone in children with CDD is a significant milestone for both Marinus and the CDD community,” said Joe Hulihan, M.D., Chief Medical Officer of Marinus. “Our ability to identify and rapidly enroll qualified patients into this study is indicative of the significant need for a new therapy to reduce seizure burden and improve patient outcomes. The study has been well conducted and we are encouraged by the limited adverse events, low dropout rates and the vast majority of patients entering the open-label extension. We are appreciative of the patients and broader CDD community for their participation in the Marigold study and we remain committed to addressing the unmet need of these patients who currently have no approved treatment options.”

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2019 AES Thought Leader Theatre

Thought Leader Theatre

IFCR President, Karen Utley, joins Eisai host Kimberly Bottom for the 2019 AES Thought Leader Theatre at the 2019 American Epilepsy Society Meeting to talk about our organization and CDKL5 Deficiency.

Read more to watch the video!


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CDKL5 Deficiency Receives ICD-10-CM code: G40.42

ICD-10-CM code for CDD Approved!

Together with the Loulou Foundation and the directors of the CDKL5 Centers of Excellence, we submitted a proposal for the creation of a new ICD-10-CM code for CDKL5 Deficiency Disorder (CDD) that was reviewed during the September 11 meeting and is currently receiving comments until November 8, 2019. It is a pleasure to announce that the application to create a new ICD-10-CM code for CDD: G40.42 Cyclin-Dependent Kinase-Like 5 Deficiency Disorder, CDKL5 was approved.

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CDKL5 Forum 2019

The CDKL5 Forum is an annual “by-invite-only” event hosted by the Loulou Foundation to report on the progression of the CDKL5 Deficiency Disorder field. IFCR board members, Rick Upp and Amanda Jaksha, and Science Director, Heidi Grabenstatter, attended this event that includes scientists, industry representatives, clinicians, and nonprofit leaders invested specifically in CDKL5 Deficiency Disorder (CDD). It was clear that the rapid progress occurring in CDD research …

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IFCR Launches Two New CDKL5 Centers of Excellence

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.

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CDKL5 PFDD Webinar

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.

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2019 NORD Living Rare Forum

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 …

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