Please visit www.hopeformarian.org to donate for research for a cure and for more information.
Please visit our new site www.hopeformarian.org to donate and for more information about Marian and The No Pucker Challenge.
While we initially began Marian's story on GoFundMe, Hope For Marian is now a non-profit in the making and can accept tax deductible donations at www.hopeformarian.org. 100% of donations will help fund research to find a cure for NPC and most importantly help save the life of a child with NPC. Thank you!!
We are asking for your help to save our sweet little girl, Marian. Last year, Marian underwent extensive genetic and clinical testing after her doctor detected an enlarged spleen. To our devastation, on February 28, 2017 we received the results of a genetic test and learned she is in a fight for her life against a fatal storage disorder called Niemann-Pick Type C (NPC). She is only 22 months old.
The Disease and Devastation
NPC is a heartbreaking rare disease often referred to as "Childhood Alzheimer's." It is a recessive genetic condition that causes a buildup of cholesterol and other material in cells, eventually leading to organ damage and irreversible trauma to the brain. As it progresses, NPC causes dementia and the gradual but complete loss of the ability to move, eat, speak and breathe. Without intervention, half of all children diagnosed with NPC will die before the age of ten.
An Effective Treatment
Despite her dire diagnosis, Marian has hope on the horizon to extend her life for a cure to be found. A clinical trial drug, VTS-270, appears to stall the progression of NPC, giving critical time to live. Currently, VTS-270 is in a phase 3 clinical trial and is administered to Marian via spinal tap under general anesthesia every two weeks, for the rest of her life.
One of the youngest patients in the world to do so, Marian receives VTS-270 through a compassionate use program run through Rush University in Chicago. After three months of flying to Chicago every two weeks to receive the treatment, Marian began treatments in June 2017 at our local California hospital, Children's Hospital Los Angeles. These past months have been a miracle to see Marian come to life again. But it is all bittersweet as we know her life is still on the line.
We Need Your Help to Find Additional Treatments and a Cure
Thanks to medical advances being made, Marian is very much a healthy and happy 22 month old today. Her young age and early intervention give her a tremendous fighting chance to survive. Labs across the country and world are developing other promising therapies for NPC as well as potential cures. Scientists at Washington University in St. Louis, Albert Einstein College of Medicine, Univesity of Pennsylvania, the National Insitute of Health and more are working tirelessly to cure NPC. However, time is finite as are research funds. If NPC progresses more quickly than the science, we will lose Marian and the other children fighting NPC today. Painfully, we could lose the battle by mere months.
By raising awareness and money we can help fund and find additional treatments and the cure in time to save Marian and others with NPC. 100% of donations through the Hope For Marian Foundation, now a 501(c)(3), will go to advancing medical research, supporting patient access, and directly to laboratories working on accelerated and directed research towards a cure.
Please join us as advocates for NPC and to help spread awareness. Please share Marian's story and #hopeformarian with your friends and networks to help us build awareness, support and the critical funds that will be needed to save the lives of children with NPC.
Together we can make a difference - for Marian and for all children with NPC. While we cannot know all the future holds, we are certain that with your support the future will include Marian.
Thank you from the bottom of our hearts,
The McGlocklin Family,
Sara, Paul, Emily and Marian
Together We Can Cure NPC
- Kyle Clark
- Lorraine Blanski