Not long ago, I found myself speaking to a program administrator at the FDA. It was a classic elevator pitch, I told him all about the breakthroughs in gene therapy for leukodystrophy, specifically, MLD. My voice cracked with emotion recounting how I had recently told one father that while gene therapy had shown promise for other children, it would not be available to his daughter. I pleaded with the FDA program officer, "What would you have me do?" The man leaned over and wh

This summer, Children's Hospital of Philadelphia social worker Sarah Stoney and Cal's Foundation co-founder Maria Kefalas were discussing how difficult it was for families to explain leukodystrophy to children. A year earlier, a school teacher emailed Kefalas to ask if there were any resources to talk about leukodystrophy for the classmates of a recently diagnosed child. "It was embarrassing to say we did not even have a brochure." So Kefalas and Stoney decided to do somethin