Patient Stories

“There’s a lot of gratitude — for my care team and the advancements that made this transplant possible, and for the donor and their family who gave me this second chance. I carry that with me every day.”

“She’ll walk up to someone — even an adult — and start a conversation. She can’t wait to go to school or the center each day.”

“Since gene editing therapy, I’m able to go to work, attend school and come home to my son without worrying about health-related disruptions.”