When the Food and Drug Administration recently approved the first gene therapy to treat metachromatic leukodystrophy, a fatal childhood inherited brain disease, Maria Kefalas was not bitter that the treatment did not arrive sooner to save her daughter, she writes for The Philadelphia Inquirer.

“For me and other families of children who have MLD, the news of the FDA’s approval of the new treatment, Lenmeldy, is cause for rejoicing,” she said.

Kefalas and her husband learned their daughter had MLD, which destroys the brain’s white matter and renders kids paralyzed, nonverbal, and dependent on a feeding tube, in 2012.

At the time, no treatment was available. A year later, Milan researchers announced they had successfully used gene therapy to treat three babies with MLD.

“Even as I watched my darling daughter slowly die, I found my purpose: I established a foundation in her honor and began raising money for MLD, one cupcake sale at a time,” said Kefalas, professor at St. Joseph’s University.

Over a decade, the Bala Cynwyd-based Calliope Joy Foundation managed to raise almost $1 million. That money was used to send 20 children to Italy where they received gene therapy for MLD.

Read more about the Calliope Joy Foundation’s impacts on finding a treatment for this rare disease in The Philadelphia Inquirer.

Donation Matching Cupcake Gala 2022: A Word from our Founder | The Calliope Joy Foundation