That is the news we received for two of our beautiful daughters, Kinley (7) and Kennedy (4), the morning after our third daughter, Kieran, was born. Without warning, we were faced with the sobering realization that our baby girls are dying. Before this disease takes them, Kinley and Kennedy will very slowly lose their ability to walk, talk, eat…and even smile.
We have been on a 4 year journey trying to understand what might be causing the developmental delays Kinley has been experiencing. After countless appointments and evaluations with specialists from nearly every medical field, we finally received our answer on June 5th 2020 - the one we never saw coming. A full genetic screening revealed that not only Kinley, but her younger sister Kennedy both have an extremely rare genetic disorder called GM1 Gangliosidosis. We are forever thankful that baby Kieran is not affected by this wretched disease.
Currently, there is no approved treatment or cure for GM1, and it is always fatal in children.
OUR NEW REALITY
GM1 Gangliosidosis is an aggressive disease where the body does not produce adequate enzymes to break down certain molecules. Instead, these molecules collect on the brain and spinal cord, leading to widespread neurodegeneration. The list of complications that our girls face is truly terrifying and the idea of watching them slowly fade away is our worst nightmare.
Right now, Kinley and Kennedy are in a relatively “stable” state in their disease progression. They can still walk independently and are verbal, though both are experiencing speech issues that make them hard to understand. Looking at them, one would never know they both face such a grave fate, but there will come a day where each of them will no longer be able to run outside with their friends, jump on their trampoline, or tell their baby sister “I love you” if no treatments are developed.
Kinley, who wants nothing more than to be a doctor, nurse, or vet so she can help other people, will eventually not be able to take care of herself independently.
Kennedy, who loves to dance and sing and dress up, will someday not be able to do her infamous twirls and leaps, or “freeze” you with her Elsa powers.
Each morning, we wake to the reality that we are another day closer to that outcome. One day, they won’t recognize their mom and dad. One day, they won’t be able to tell us they need something, or that they love us. One day, they will require a feeding tube. One day, we will lay two of our precious babies to rest.
Life expectancy for the type of GM1 Kinley and Kennedy have is difficult to predict, but estimates range anywhere from mid-childhood (10-15 years old) to the early twenties. We are hopeful that both Kinley and Kennedy have many more great years ahead of them at a quality of life that they both deserve. There is no way to know at what point the disease will firm its grip on Kinley and Kennedy - we are in a race against time.
SOME HOPE - IT'S NOT TOO LATE
There are several treatment clinical trials coming up for both substrate suppression therapy and gene therapy through drug companies, universities, and the NIH. This Page is a mission of hope for our family and others impacted by GM1 Gangliosidosis, and provides support to ensure these trials and developments continue. We are committed to raising awareness of this horrid disease, and promoting funding for continued research and the advancement of current clinical trials that may eventually be accessible to these children, including our own. In fact, some believe a cure is imminent - it simply needs the support and dollars to advance. We are not naive to the fact, however, that clinical trial spots are very, very limited and are experimental, so there are never guarantees...but we do know that our girls do not need to suffer!
We intend to soak in every moment with Kinley and Kennedy while they can still run, dance, sing, and just be kids. We now know how precious each and every day is, and appreciate every time we hear the words “I love you” from them, or hear them sing lullabies to their baby sister. While continuing to give our girls all the love we can, and show them the world as best we can, we simply must fight to find off-label treatments to slow the progression long enough for a cure to be developed.
We will still show them the world, provide experiences even seasoned adults have never had, and make sure every moment they have on this earth is not taken for granted.
Developing treatments in the United States is notoriously expensive. For gene therapy specifically, it is estimated to cost between $1,000,000 and $2,000,000 for a single clinical trial participant*.
We are raising $2,000,000 - the conservative estimate for both Kinley and Kennedy to participate in a gene therapy trial. We refuse to sit idly while our babies bodies slowly betray them and we can only achieve this goal with your support. Every dollar is immensely important, and sharing the information with your network can and will work miracles. Reaching this goal won’t guarantee Kinley and Kennedy treatment, but it will go far in advancing the development of the cures we and families in our position so desperately desire.
We all say “we will do anything for our children”. We’re asking for you to help us in this journey so our children and other families in our position can retain hope that there is a better future for our babies.
SPECIFIC WAYS TO HELP
1) If you’re in a position to do so, please consider a donation. Every dollar counts.
2) Spread the word - share to your social networks, email the GoFundMe information to anyone you know, and be sure to ask if your company has a donation matching program!
3) AmazonSmiles - if you aren’t already supporting a charity of choice, please take a moment to update your AmazonSmiles beneficiary to Cure GM1 Foundation. After that, shop as you normally do, and a donation will automatically be provided for each dollar spent.
4) Forward any leads you may have to support our cause to our Facebook Page or our email address:
WHERE IS THE MONEY GOING?
Every dollar will benefit the Cure GM1 Foundation, a 501(c)(3) nonprofit organization whose mission is to advance the development of life-saving treatments for GM1.
The Cure GM1 Foundation is currently funding a next generation gene therapy program in collaboration with a world class research team. The team is headed by Dr. Krystof Bankiewicz at Ohio State University who works in close collaboration with Children's Nationwide Hospital in Columbus, Ohio. Other initiatives include funding research studies to further drug development, newborn screening, and increasing overall awareness.
All donations are 100% tax deductible!
*“Novel Financing Approaches Are Needed to Capitalize on Life-Saving Gene Therapies∗.” The American Consumer, The American Consumer Institute Center for Citizen Research, www.theamericanconsumer.org/wp-content/uploads/2019/02/Gene-Therapy-FINAL.pdf. Accessed 1 June 2020.
- Sara Hahn
- Memory of Bob and Judy Cooper
- Amanda Mrkvicka
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