Lori Sames, co-founder of Hannah’s Hope Fund, which supports research to find a cure for Giant Axonal Neuropathy (GAN), was honored by the National Organization for Rare Disorders (NORD) by having her story told as part of their 2015 Portraits in Courage celebration.
When Lori Sames’ daughter was born with GAN, a rare disease that causes progressive nerve death, she and her husband, Matt, discovered that there was only one researcher in the world studying GAN. They decided to take action by starting Hannah’s Hope Fund to help find a cure. Five months later the first symposium on GAN was held. Now seven years later, the world’s first spinal cord therapeutic gene treatment is underway to see if a gene transfer treatment can help people with GAN.
Dr. Steven Gray, member of the UNC Gene Therapy Center and Assistant Professor of Ophthalmology, spearheaded all of the studies to initiate this Phase I gene therapy trial for Giant Axonal Neuropathy and is an associate investigator in the ongoing Phase I clinical trial being conducted at the NIH Clinical Center.
For more information about the clinical trial, “Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy,” visit the clinicaltrials.gov website.