Please nominate Hannah’s Hope for the NY Yankees Hope week. (It takes less than 5 minutes.) Click the “Nominate Now” button on the NY Yankees Hope week page.
Here’s some useful information to help you complete the form:
HHF’s city is Rexford.
HHF’s zipcode is: 12148
You can cut and paste the text below for the “Nominee Description” section:
Three years ago, Lori and Matt Sames of Rexford, NY received the worst news any parent could hear. When a geneticist explained to the Sames that their 3-year-old daughter, Hannah, had Giant Axonal Neuropathy (GAN), an extremely rare and fatal illness, Lori asked, “So you are telling us this is a death sentence?” The geneticist replied, “Yes.”
GAN slowly takes away a child’s ability to walk, use one’s hands, speak, swallow and is terminal usually by the time a child reaches their twenties. The disease is caused by mutations in the GAN gene which is involved in a cellular function that gets rid of excess proteins. In children with this disorder, the excess proteins build up, eventually resulting in problems with movement and other nervous system dysfunction.
“Hearing Hannah’s diagnosis was the single most devastating moment of our lives,” said Lori. “For a couple of days, we walked around in complete devastation. And then Matt said to me, ‘Someone has to be the first to be cured.’”
“It is your worst nightmare,” said Matt Sames, Hannah’s father. “They gave us shattering news about our baby, and then we found there was nowhere to go for information, research or support.”
GAN is so rare that there are less than 50 known cases world-wide. When the Sames first learned about GAN, they had no way of contacting other families coping with the illness.
The Sames were unwilling to accept the “death sentence” diagnosis and decided Hannah would be the first to beat the disease. They created Hannah’s Hope Fund (HHF) for GAN, a 501c3 non-profit organization, the only in the world dedicated to finding a cure for GAN.
Since its inception, HHF has made remarkable strides and is less than a year from beginning a gene therapy clinical trial to treat GAN. HHF assembled a team of scientists lead by the University of North Carolina at Chapel Hill Gene Therapy center who advanced a therapy which will have applicability to other diseases such as Lou Gehrig’s disease (ALS) and Spinal Muscular Atrophy (SMA).
HHF, primarily a family and volunteer driven charity, is working tirelessly to move Hannah and all the children suffering from GAN one step closer to receiving a treatment that will stop the progression of this disease and ultimately save their lives.
GAN is an “orphan disease,” defined as a very rare disease for which there is little or no resources available. Because it is an orphan disease, there is no funding for research other than what HHF has provided. Since Clinical Trials are experimental, they are not covered by insurance. Research and FDA required studies cost hundreds of thousands of dollars and a clinical trial for one child is estimated to cost approximately $25,000.
Thanks to HHF, the families of GAN are no longer alone in their struggle. HHF helps provide the Miracle of Hope for these children and their families.