Iris is a strong and sweet girl. She wrangles with her little brother Carter on a regular basis, as any big sister should. She has a beautiful smile and a contagious giggle. She loves riding and swim therapy and attending school.
In August 2013, our family experienced every parent’s worst nightmare. Iris was diagnosed with a terminal degenerative neurological condition called Juvenile GM1 Gangliosidosis. We were completely blindsided. We had been told that Iris’ development was normal, despite troubles with speech and her motor skills. The progression of the disease is now clear. We fear the impact of the condition ranging from seizures, blindness, deafness, and the inability to eat, speak and move.
The only suggested medicine that exists may possibly slow the disease, if that. It is not even FDA-approved for this specific disease. Many people have tried the medication with limited success or they experienced no benefit at all. There are no treatments for this condition other than comfort measures and symptom management.
GM1 is progressive. The disease worsens with time. We do not know what the future holds. We hope Iris will live many more years, perhaps into her teens or even her twenties. But we do not know how her quality of life will be maintained. If there were a choice between quantity or quality, what would one choose? 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 to 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 sweet girl.
Our goal is to raise awareness and funds to support scientific and medical research for GM1 Gangliosidosis. We want to to help all those who suffer from this disease.
We believe a cure is within reach given adequate resources.
Research and Possible Cures
Research is advancing on multiple fronts globally. These efforts require financial support.
At this time, our fundraising efforts have resulted in 4 research projects. And research we funded is approaching human clinical trials, but still requires funding for drug manufacturing and running the trial itself. We are aware of many extremely promising research projects, but none have made it to humans.
There is a phase in drug development and research called the valley of death. This is when drugs and research projects enter a phase where all progress ceases due to lack of funding.
We must contribute in any way possible to bring these treatments forward.
Help Sweet Iris and Donate
We as parents are advocates for our children. We strive to protect our children. We make every possible effort to shield them from 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. Rare diseases and this category of conditions afflict far too many people.
Every second that goes by, we could be closer to an end to GM1! All donations, small or large count towards raising funds. All kind thoughts and prayers are appreciated.
THANK YOU from the bottom of our hearts for reading and to anyone who is willing to help us in this enormous fight for life!