Jenny's GM1 Journey.org wrote -
All funds go toward the goal of the non-profit 501(c)(3) Cure GM1 Foundation (EIN #47-3154419), which is advocating for a cure or treatment options for children with GM1. All donations are tax deductible.
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Cure GM1 Foundation
PO BOX 6890
Albany, CA 94706
Reference: Jenny's GM1 Journey
Our lovely daughter Jenny is 12 years old and in 6th grade. Like any big sister, she plays and teases her little sister Jamie on a regular basis. She has a beautiful smile, contagious giggle, and loves to give hugs. She loves nature, dolphins, and to draw.
In August 2014, after 8 years of pursuing medical diagnosis our family experienced every parent's worst nightmare. Jenny was diagnosed with a terminal degenerative neurological condition called Juvenile GM1 Type 2 Gangliosidosis.
We were completely stunned, up until that time medical experts had not perceived her medical condition to be that serious. In her early years we had been told that Jenny’s development was slightly behind, despite troubles with speech, physical capabilities, and her motor skills. Over the past several school years, clear evidence has shown the disease progression which has had serious impact to her learning, development, and ability to interact with peers.
Based upon research by the University of Minnesota and NIH, the only medicine under study is “miglustat”, a treatment that has shown some benefit to slowing the disease’s progression. It is not a cure. There are no other treatments for this condition other than comfort measures and symptom management. GM1 is also not included on the compassionate allowance list to receive Medicaid support.
GM1 is progressive. The disease, if left untreated, rapidly worsens with time leading to eventual death. While we do not know what the future holds, we hope Jenny will live many more years, perhaps even into her twenties. But we do not know how her quality of life will be maintained. We know the inevitable course this disease takes. There are no guarantees.
We cherish every moment and we take nothing for granted. Each day is a new beginning. We are hopeful that somehow we can contribute and find a cure. However, we are fighting in a race against time. This disease is not specific to any race, ethnicity or individual group. We are a typical family with a very grave concern. We have an enormous amount of love for our beautiful daughter.
The Condition and Prognosis
GM1 Gangliosidosis robs children of nearly every skill and bodily function required to live. An insufficient amount of the enzyme beta-galactosidase is produced by the body. This deficiency causes a breakdown in the processing center of cells. This dysfunction results in cell death, particularly in extremely fragile neurons and spinal cells.
Many of the children lose the ability to speak, to swallow, and to move their limbs. Some become blind and deaf. The list of possible complications is truly scary and extremely long. The brain is the control center for the body. The body cannot function properly with a brain under attack. Seizures eventually consume the mind and body.
Many of the children succumb to pneumonia due to weakened immune systems and respiratory complications after a prolonged fight for their lives.
Our goal is to raise awareness and funds to support scientific and medical research on the cure for GM1 Gangliosidosis. It is estimated 1 in every 200,000 children may be afflicted and we want to help all children who suffer from this disease.
Based on our research shared by the Cure GM1 Foundation and several leading medical research universities, we believe a cure is within reach given adequate resources.
Research and Possible Cure
Research is advancing on multiple fronts globally. These efforts require financial support. In particular, our goal of this campaign is to raise funds for the Cure GM1 Foundation to support gene therapy research at University of Massachusetts Medical School (UMass), University of Minnesota (UMN), Auburn University in Alabama (AU), and get the cure through FDA approval to clinical trial.
The therapy being developed by Auburn University and UMass may cure children in a single treatment and it needs to get to human clinical trials.
We must contribute in any way possible to bring these treatments forward.
Help Jenny and Donate
As parents, we are advocates for our children. We strive to protect our children. We make every possible effort to comfort them while pain and suffering. We do not want to question "What if we had asked our friends, families and the world for help? Could we have done more?"
Every moment that passes is one step closer to further degeneration. This disease is relentless and cruel.
All kind thoughts and prayers are appreciated however action is needed. Every minute that goes by we could be closer to an end to GM1 or better treatment!
All donations, small or large, count towards raising funds for the cure. All donations are a Hug for Jenny!
- Share our CrowdRise Campaign and encourage everyone you know to donate.
- Please help this campaign grow so it may contribute to saving Jenny and GM1 children for generations to come.
THANK YOU from the bottom of our hearts for reading and to anyone who is willing to help save Jenny!