One Shot To Live – There is just one shot…

RARE Village Presents

synopsis

Single-gene therapy may be the greatest hope for thousands of children with rare diseases. But the greatest challenge is time, or the lack of it. In an increasingly faster-paced world, the parents of these three children fight to save lives all while seeing the life around them through an entirely new lens; a lens that lets them live each day to the fullest.

“I really want to help make a fundamental difference in families’ lives by altogether eliminating the problem in as many cases as we can.”

- Dr. Berge Minassian

"Do I want to be doing science for the sake of doing science, or do I want to apply it to a directed cause? And I think the connections I've made with some of the families and the kids made it crystal clear to me that I knew which way I had to go."

- Dr. Steven Gray

"It's the most unfair thing in the world," said Gray. "If the parents don't become advocates, and if they don't become funders, then it's just not gonna happen."

- Dr. Steven Gray