After Gordon Gray’s daughters Gwenyth and Charlotte were diagnosed by doctors with the rare neurodegenerative Batten disease, they were told there was nothing to be done except prepare to watch their daughters go blind, lose all motor & verbal functions, the ability to feed themselves, and inevitably death. The Grays began rallying friends and family to find some way to arrest the development of this disease.
A year later, the girls were participating in a clinical trial of the first gene therapy of its kind, where she was given a virus infused with the healthy CLN6 gene, allowing cells to rid waste build-up and restore balance in the brain. Younger sister Gwenyth was given a preventive dose. Since Charlotte has shown sign of improvements.The gene therapy program was initiated, coordinated, and funded by the foundation, resulting in an FDA-approved investigational gene therapy clinical trial for children with Batten CLN6. However, the road to a cure has just begun.
Learn how you can help find a cure for Batten disease here.