Predicting the future is a messy game, but “Back to the Future Part II” got a lot of things right (while we don’t have “Jaws 19,” we have plenty of big-screen TVs).
I was 7 years old when Marty McFly and Doc Brown traveled to Oct. 21, 2015 to save McFly’s kids. I dreamed about flying cars and robots and wondered how Nintendo managed to fit so many pictures on a single 8-bit video game cartridge. continue reading →
Today, UNC Health Care announced an innovative, gene transfer-based treatment approach for children with giant axonal neuropathy (GAN). The treatment, developed by researchers led by Steven J. Gray, PhD, is the first of its kind. A clinical trial is now underway at the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH).
This is significant news for Taylor’s Tale and the Batten disease community because we’re funding Dr. Gray’s work on infantile Batten disease – work modeled after the GAN research that is now treating children at the NIH. continue reading →
Every time I visit Steve Gray and the University of North Carolina Gene Therapy Center, I want to run home and immediately write a blog post and email all of my media contacts and friends in the rare disease community and tell them what’s happening in a lab two hours from my hometown. I want to tell them Steve and his team are going to change the world.
But when I get home, I can’t find the right words, and instead I just scroll through my pictures and reread my notes and wonder how I can possibly feel so inspired knowing we’re too late to save my little sister. continue reading →
Nearly five years ago, when my sister could still sing and talk and walk and eat ice cream cones on hot summer days, my mother met Steve Gray, a young investigator from the University of North Carolina Gene Therapy Center, at a conference in Bethesda. Since 2008 he’d been working on giant axonal neuropathy (GAN), an ultra-rare, fatal childhood disease that causes progressive nerve death.
A few months later, we drove to Greensboro to have dinner with him. We weren’t ready to take the leap then, but Mom believed in Steve since the first time she heard him speak about his effort to save children from a monster that turned them into quadriplegics unable to eat or breathe on their own. When I sat across from Steve in our booth that night, I believed in him, too. continue reading →