Friends and Family-
Our darling Baby Violet, the light of our lives, has been tragically diagnosed with a life-limiting genetic disease known as GM1 Gangliosidosis.
For the last year, we have noticed that Violet has started to miss important developmental milestones, and after the onset of seizures in December 2020 we embarked on an exhaustive diagnostic work-up, which included multiple hospital visits, MRI scans, blood work, and genetic testing. In May 2021, we received the devastating news that Violet has GM1 Gangliosidosis, a rare progressive genetic disorder that affects the central nervous system.
Currently, there is no approved treatment or cure for GM1, and it is always fatal in children.
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 neurologic degeneration. Life expectancy varies, and can range from early childhood to early adulthood.
We have been overwhelmed with the outpouring of support in our community, and by the wonderful affected families who have reached out to us to share their stories and experiences. It has been truly the most difficult thing that we have ever had to deal with, and in the center of it is our darling daughter who is starting to show evidence of the disease. Soon she may lose the ability to see, hear, and even speak.
But in this tragedy we have found a bit of hope. The CureGm1 Foundation was founded to fund critical medical research and development for GM1, and in the last year we have seen actual human clinical trials with promising results. Your donation can help fund clinical trials and expedite research for a cure. We are cautiously hopeful that Violet may be a candidate for some of these therapies in the future; if so, it could possibly alter the natural course of this disease and give her a chance at life.
We are aiming to raise $50,000 for the CureGm1 Foundation for Violet's second birthday, which is June 11, 2021. Through advocacy and fundraising, we truly hope to make a difference in all children affected by this disease. Already, the efforts of this wonderful organization have resulted in research and clinical trials which are already making a difference. With your help, we can potentially cure this devastating disease. And cure our darling Violet.
A video of Violet's progress can be found here: https://youtu.be/m71NLBnFM_Y
Thank you for your time and your help. We appreciate everyone's support and will update you on Violet's progress. Thank you!
-David, Veena, and Violet
More information about Cure GM1 Foundation: Cure GM1 is the only 501(c)(3) dedicated to funding medical research for GM1 Gangliosidosis. GM1 is always fatal in children.
Why do we fundraise? Because changing the future of a disease is massively expensive.
* A single mouse model can cost $150K
* A single manufacturing run for plasmid for gene therapy costs $350K+
* A small clinical trial, i.e., excluding cost of drug can cost ~$3M+
* A manufacturing run for gene therapy vector costs ~$1M-$1.5M.
Every little bit helps. We continue to push forward on multiple fronts to assist in advancing research and to ultimately help reach patients and families who are desperately seeking a possible treatment.
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