Our Pledge to Patients

With patients as our focus, we are continuously seeking ways to position ganaxolone within the treatment paradigm of rare epilepsies and neuropsychiatric disorders.

Our dedicated team of professionals, scientists, and medical experts strive to demonstrate the value of ganaxolone as a potential first-in-class, fast-acting, safe, and innovative therapy for children and adults afflicted with disorders affecting the central nervous system.


Patient resources and clinical trial information


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Status Epilepticus (SE)

Status epilepticus is the occurrence of an epileptic seizure of a prolonged duration of more than five minutes or several seizures within a five-minute period where the individual does not recover between seizures.


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CDKL5 Deficiency Disorder (CDD)

CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome.


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PCDH19-Related Epilepsy

PCDH19-related epilepsy is characterized by highly variable early-onset cluster seizures with comorbid cognitive and behavioral disturbances with or without intellectual disability.


Icon Tuberous Sclerosis

Tuberous Sclerosis Complex (TSC)

Tuberous sclerosis complex (TSC) is a rare genetic disorder that affects many organs and causes non-malignant tumors in the brain, skin, kidney, heart, eyes, and lungs.

Collaboration

We collaborate with patient advocacy organizations to increase awareness and education, promote earlier diagnosis, advance new therapies, better understand the patients we serve, and improve quality of care. Together, we hope to make a difference.

If you are a patient advocacy organization interested in collaborating with us, please email Romy Nocera : rnocera@marinuspharma.com

CDKL5 is painful. It’s a hard, sad at times, thing that we face. When you have a relationship with people like Marinus and their researchers, you are able to help be a driving force behind that work.

Karen Utley, Mother to Samantha, President of International Foundation for CDKL5 Research

Marinus has been wonderful in that they are very proactive in working internally with U.S. based clinics that we work with and have the infrastructure to do clinical trials. One of the most difficult things is to navigate different healthcare systems and Marinus took that on and made it happen so that all of the different communities could participate.

Heidi Grabenstatter, Science Director at International Foundation for CDKL5 Research

My involvement with Marinus has always been based on my belief that they are the most patient-centric pharmaceutical company. The fact that they’re willing to share the voices of CDKL5 parents and their children shows their commitment.

Carol-Anne Partridge, Mother to Amber, Secretary of CDKL5 Alliance, Chair of CDKL5 UK

The first company that chooses to run a clinical trial for a rare disease is always a very unique company and special partner for the patient community. Most companies would stay away from a risky disease. When one company decides they’re willing to take a risk, they’re essentially building the field for others. In the CDKL5 community, that special company is Marinus.

Ana Mingorance, Chief Development Officer at LouLou Foundation

As a community, I truly believe we are being heard by the researchers and pharmaceutical companies like Marinus who are exploring treatments for CDKL5. That’s a very empowering feeling as a mom knowing I am making a difference.

Jonna Malone, Mother to Maya, President of CDKL5 Ireland

Because we are able to work together, things like clinical trials are made possible. Lives are changing, kids are able to enroll, our children might have less seizures — that is huge.

Julie Walters, Mother to Violet, President of PCDH19 Alliance

PCDH19 is an extremely complex condition. I am thankful Marinus is dedicated to improving the quality of life for children.

Jennifer Holland, Mother to Sloan

It means a great deal to have companies that are willing to do trials and research for TSC because TSC is a lifelong journey. I am grateful for the companies investing in research to give my kids a brighter future.

Shannon Grandia, Mother to Rylee, Jake, and Luke, TS Alliance

Folks like those working at Marinus really understand patient needs and if they are focused on the unmet patient needs, we are going to make good progress together.

Dr. Steve Roberds, Chief Scientific Officer at TS Alliance
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