Giant Axonal Neuropathy (GAN)
WHAT IS GAN:
Giant Axonal Neuropathy (GAN) is a recessively inherited condition that results in progressive nerve death. GAN generally appears in early childhood and progresses slowly as neuronal injury becomes more severe. As the disorder progresses, patients become quadriplegics, dependent on a feeding tube and ventilator before dying typically in the second or third decade. However, we now have some confirmed GAN cases that are milder progressing, and also have confirmed cases with straight hair. The doctors working with Hannah's Hope Fund now suspect that CMT Type 2 patients, whose causal gene has not yet been identified, may have GAN. Hannah's Hope Fund has a collaborator for GAN gene sequencing. For more information, visit the Doctors and Families page.
Hannah’s Hope Fund for Giant Axonal Neuropathy, Inc. is a 501(c)(3) public charity whose mission is to raise funds to support the development of a treatment and cure for GAN, and to be the resource for doctors, scientists and families world-wide.
HOW YOU CAN HELP:
Donate Now or join the many GAN families across the Globe and help us raise funds for a treatment and cure which will save their precious lives. Together these families have all come together to help save their children but they need your help. Help them raise funds towards the continued development of the gene therepy and a therapeutic drug approach. Help Hannah's Hope Fund spread awareness so that other families will know they are not alone.
The GAN Gene Delivery Clinical Trial is Officially Posted on the National Institute of Health's Website and is recruiting participants!
Click to view the GAN Clinical Trial page.
The following article appeared in the Albany Times Union:
By Claire Hughes, Published 11:10 pm, Friday, February 13, 2015
Hannah and Lori Sames
Excerpts from the article follow. Click to read the full article.
"Tears were rolling down my cheek," Sames said. "Tears of joy that finally there's an experimental trial that's active for this disease..." Such a study has been the dream of Sames and her husband, Matt, whose 10-year-old daughter Hannah suffers from GAN...Hannah's Hope Fund has supported one study after another ... through $6.5 million raised from the foundation's headquarters in the Sameses' Rexford kitchen. (Their bare-bones overhead means about 97 percent of donations go to research, Sames said.)
The clinical trial is not for healthy volunteers, but only those with GAN, at least 5 years old. Participants must live for two months within 100 miles of NIH in Bethesda, Md., undergo a battery of medical tests and be monitored over 15 years. They will have a gene, packed in a modified virus, injected by spinal tap.