
"Don't give up"
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

Loie's Disease children's book provides a resource to families across the globe
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