Hope For Marian
$76,759 of $150,000 goal
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 will go to advancing medical research. All donations will be forwarded to the Parseghian Medical Research Fund, a 501(c)(3) charitable organization, 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
Together We Can Cure NPC
We've been very lucky to connect to each of you who are our circle of support helping to lift Marian and NPC closer to a cure. We are overwhelmed with gratitude and thank you from the bottom of our hearts. Please continue to follow her journey and check out progress towards a cure at www.hopeformarian.org. We can do this (I'm at least 90% sure).
Sara and Paul
We had no idea whether she could get the treatment, whether it would help her, and for how long (it doesn't work forever).
On this day my cousin's sister and incredible friend Andrea called. Her boss and his friends wanted to donate their time, talent and hearts to make an awareness video for Marian/NPC. They aren't PR people. We haven't paid them a cent. They are amazing. They are business owners who want to do good and help. They are West Point grads and veterans who are fighters, and they are now fighting for Marian.
The campaign they developed is powerful and has touched our hearts and lives, and we think it will touch yours as well as members of Marian's team for hope and life. A powerful team we are deeply grateful for.
Today, Marian can walk, she remembers the words she forgot, she can stand. She's learning new words, is full of energy and laughter (and even some mischief). SHE is fighting. This campaign is a huge gift that fell into our laps - and it has the potential to save her life. Will you help us spread this campaign to bring more treatments here faster and in time for Marian?
Here is how you can help:
1. Please watch and share the Marian story video, there will be more to come
2. Please watch The No Pucker Challenge and please make your own No Pucker Challenge video for Marian! Eat a lemon, or a big bite, to help cure NPC. Nominate three friends. And try not to pucker! Share with #hopeformarian #nopuckerchallenge #cureNPC. Please get your kids, pets, work or friends involved if you can!
3. Visit www.hopeformarian.org where we are able to accept donations and are forming a 501(c)(3) to donate, get more information about this campaign, and buy a shirt.
4. Buy a Hope For Marian shirt - they're super soft, adorable and we are so proud of them!
You all kept us standing and opened the window of hope for Marian, we really believe together this can spread across the country and world to help save Marian's life and other children and adults fighting for their lives against NPC.
Sara, Paul, Emily and Marian
We've made four round trip visits to Chicago for treatment. Our next one is June 9th and we are hoping it will be the last biweekly trip. CHLA and Chicago have both been working hard to get her treatments set up locally and it's getting closer, which is very exciting. Marian amazes me each time with her inner resolve and strength, she has a deep fight in her along with a heart bursting with love.
On the medical front, all is well. Her dose was raised up one level this last time which she handled well. Marian's hearing is being closely monitored as the medicine will cause high frequency hearing loss, letters like S, T, F, H and possibly more. So far so good and Marian's hearing is doing swimmingly.
Marian has been lighting up the room with her big laughter and trying to make us laugh with her jokes, her favorite is slurping out of her cup.
Paul and I will be traveling to AZ this weekend for a scientific research conference for NPC which we are looking forward to attending. We've also made our first donation with the GoFundMe money to the Million Dollar Bike Ride for Team NPC, we made a $1,000 donation which was matched dollar for dollar by UPenn and will go directly to an important NPC research grant.
We would never be able to get Marian the care she needs and keep going in the daily grind without your support and we thank you greatly! Hopefully we will be providing exciting updates soon as our journey continues. Thank you for following along with us and being by our sides.
Sara, Paul, Emily and Marian
I'll be posting more updates on Facebook and www.hopeformarian.com as things happen. Thank you so much for your support of Marian and NPC!
I'm so happy to see little Marian doing so well. Thank you for the updates. With love, Christina
So glad to hear that Marian is improving! I hope more blessings come your way!
I love these updates! Once I'm out for the summer I can get them more in person during some much need pool time!
Marian and your family continue to be in my prayers. I'm pleased to know Marian is reacting well to the treatments. I send you my love and prayers ♡
So happy she is doing well!!!!!!
Do your research. .CBD can help your little girl..it's helped so many people. .I wish you the best
Big sigh as I read that the first Chicago cyclodextrin treatment has been completed without complication. Lots of love from the LB Lawlers.
Our hearts go out to you and your family. Marian is in our daily thoughts and prayers, and we will pray for your strength in this fight, Sara.
We have Marian in our prayers everyday. Together we will fight this battle for our little sweet girl. God bless