Help Sweet Iris
Donation protected
Please donate to our nonprofit http://www.curegm1.org from now on.
No donation will be used for personal financial support or personal medical expenses. We seek to raise funds for medical research and a cure for GM1 Gangliosidosis.
Our Story
Our sweet girl Iris is just 7 years old and she is in first grade. 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 orcas, dolphins and to draw.
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. This school year, there is now evidence that the disease impacts her learning.
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 even into her teens or twenties. But we do not know how her quality of life will be maintained. If a choice were possible, would one choose quantity or quality? 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.
The Goal
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.
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 can not 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.
Research and Possible Cures
Research is advancing on multiple fronts globally. These efforts require financial support.
In particular, our aim is to support gene therapy research at UMass Medical School and at Auburn University in Alabama. The therapy being developed could cure children in a single treatment.
Learn more about the GM1 research at Auburn University. Auburn and UMass are collaborating to bring this research forward.
Other research may also emerge that we could also support. We are in direct contact with the team of Japanese researchers. One of the primary researchers recently tested the compounds in development on a sample of Iris' very own cells. The improvement in the cells was dramatic, but human clinical trials have not been initiated.
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.
PLEASE help! Share our web site and donate. Participate and contribute to saving our sweet Iris and many children for generations to come.
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!
www.sweetiris.org
Facebook: http://www.facebook.com/HelpSweetIris
Twitter: http://www.twitter.com/helpsweetiris #HelpSweetIris
Donations to GoFundMe are not tax-exempt.
If you would like to make a gift in excess of $14000 (You're amazing!). Please contact us through the contact form on sweetiris.org
No donation will be used for personal financial support or personal medical expenses. We seek to raise funds for medical research and a cure for GM1 Gangliosidosis.
Our Story
Our sweet girl Iris is just 7 years old and she is in first grade. 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 orcas, dolphins and to draw.
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. This school year, there is now evidence that the disease impacts her learning.
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 even into her teens or twenties. But we do not know how her quality of life will be maintained. If a choice were possible, would one choose quantity or quality? 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.
The Goal
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.
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 can not 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.
Research and Possible Cures
Research is advancing on multiple fronts globally. These efforts require financial support.
In particular, our aim is to support gene therapy research at UMass Medical School and at Auburn University in Alabama. The therapy being developed could cure children in a single treatment.
Learn more about the GM1 research at Auburn University. Auburn and UMass are collaborating to bring this research forward.
Other research may also emerge that we could also support. We are in direct contact with the team of Japanese researchers. One of the primary researchers recently tested the compounds in development on a sample of Iris' very own cells. The improvement in the cells was dramatic, but human clinical trials have not been initiated.
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.
PLEASE help! Share our web site and donate. Participate and contribute to saving our sweet Iris and many children for generations to come.
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!
www.sweetiris.org
Facebook: http://www.facebook.com/HelpSweetIris
Twitter: http://www.twitter.com/helpsweetiris #HelpSweetIris
Donations to GoFundMe are not tax-exempt.
If you would like to make a gift in excess of $14000 (You're amazing!). Please contact us through the contact form on sweetiris.org
Organizer
Christine Waggoner
Organizer
Berkeley, CA
