CDD Awareness Month

June 25, 2024

By: Christy Shafer, Chief Commercial Officer

Each June, we recognize CDD Awareness Month and shed light on a rare disease that impacts an estimated 100 newborns in the U.S. every year. Navigating the complexities of a rare disease requires a collective effort to understand its profound impact on patients, their families, and our healthcare system. At Marinus, we are committed to raising awareness and collaborating with patient communities and industry stakeholders to drive positive change and advance innovative solutions for individuals affected by rare seizure disorders.

CDD, or CDKL5 deficiency disorder, is a rare, X-linked developmental and epileptic encephalopathy characterized by refractory seizures and severe global developmental impairment. The CDD patient journey is arduous, requiring dedicated caregiver and multidisciplinary healthcare team support throughout the patient’s lifetime.

While rare, CDD is one of the most common genetic epilepsies, with seizures typically beginning within the first three months of life, and a median onset at six weeks of age. Patients living with CDD may experience a wide range of chronically debilitating symptoms, including early-onset seizures, global developmental impairment, and intellectual disability, as well as disorders related to sleep, speech, physical development, and cortical vision.

Living with such a complex disorder brings many challenges, particularly when navigating a landscape with limited treatment options. In 2022, Marinus was proud to bring the first and only FDA-approved treatment for seizures associated with CDD in patients two years of age and older to this underserved community, following completion of the first-ever pivotal trial specifically in CDD. This breakthrough alone is a testament to the endless determination of advocates and researchers in the CDD space who work tirelessly to bring solutions to those in need. Having witnessed firsthand the impact of this devastating disorder on patients and families, it is important to celebrate these victories as we strive to create a better future for families.

The key to continued success lies in collaboration, education, perseverance and advocacy. Together, let’s amplify our efforts to raise awareness, support research initiatives, and champion the needs of individuals and families affected by CDD. I would encourage readers to visit the Marinus website here, to learn more about CDD, as well as our ongoing commitment to help improve outcomes for those impacted. Additional resources are also available from some incredible patient advocacy organizations in the CDD disease space, including the International Foundation for CDKL5 Research, CDKL5 Alliance, CDKL5 Research Collaborative, Loulou Foundation, and Hope4Harper.

By uniting our voices and resources, we can drive meaningful change and pave the way for improved treatments and outcomes. As we conclude CDD Awareness Month, let us carry forward this momentum, standing alongside the CDD community in solidarity and hope for a brighter tomorrow.