When four-year-old Harlow Prado woke up from anesthesia after her first dose of life-saving gene therapy, the first thing she asked was, “Mama, can I walk yet?”

Harlow has a rare brain disorder called TUBB4A-related leukodystrophy, which affects her motor development. For her entire life, she has relied on a walker and attended eight therapy sessions a week.

But on October 15, she became the first girl in the world to receive a personalized treatment for the disorder—one that could help her achieve her dream of walking.

Speaking to Newsweek from her daughter’s bedside in the hospital, Daphne Graskewicz-Prado, from Southern Illinois, said: “I wasn’t going to accept the terminal diagnosis.”

What Is TUBB4A-Related Leukodystrophy?

TUBB4A leukodystrophy is an extremely rare and serious b

See Full Page