Why We Created the
Hope For Marian Foundation
Why We're Here
In 2017, time stood still when we learned our younger daughter Marian was in the early stages of a fatal storage disorder. She continues fighting for her life against Niemann-Pick Type C, also known as "Childhood Alzheimer's." We created Hope For Marian Foundation to raise awareness, advance research, and support patient access to treatment for all NPC patients.
Marian was born on August 26, 2015. Behind with physical milestones at nine months, we consulted a neurologist as a precaution (three different ones, in fact). You can read our full diagnostic journey here. Her symptoms were mild, but puzzling. Marian was just a year old when her neurologist at Children's Hospital Los Angeles felt an enlarged spleen. This changed our course from precautionary monitoring to bracing for devastation. After months of testing, our worst fears became reality: She has NPC. Progressive loss and suffering. No cure. Fatal.
At just seven months, Marian needed her arms to support her, her belly was swollen from an undetected enlarged spleen.
Hope For Marian and NPC
Shortly after diagnosis, we found hope and the path to survival through experimental treatment. Marian receives this critical lifeline every two weeks through a spinal tap at Children's Hospital Lost Angeles.
These early months after treatment were a miracle to see Marian come to life again. At 20 months, Marian could not take a single step. At 23 months and after eight treatments, she could very proudly walk across the room.
Focusing on supporting patient advocacy and access to medicine
Research - co-funded a multi-year research project at University of Michigan in the Mark Schultz lab which helped improve an important research model
Advocacy - organized the Save Our Medicine campaign, previously called Don't Give Up On NPC
Education - worked with the NPC community to organize a virtual Capitol Hill briefing to educate members of Congress about urgent NPC issues
Regulatory Work - began funding a FDA consultant and other experts to support patients maintaining access to experimental medicines and help ease hurdles in regulatory pathways
Support - continuous support of patients and families, working together to save access to two experimental NPC medicines - adrabetadex and arimoclomol
Access - funded grants to the Guest House in Chicago and the patient access fund at Rush Hospital
Experimental treatments are working to slow the progression of this relentless and fatal disease.
Today, patients' lives hang in the balance and are dependent on continued access to existing experimental medicines which are at risk.