Mila was once like any happy, healthy child. But at age 3, she started to falter. At 6, she was diagnosed with Batten disease, a rare and fatal genetic disorder. She lost her sight, her language and her ability to walk independently. No one had an answer — until a plea on Facebook led her parents to Dr. Timothy Yu in Boston Children’s Hospital’s Division of Genetics and Genomics.

Ed. note: Mila passed away in February 2021, at age 10. The Mila’s Miracle Foundation: https://www.milasmiracle.org/ continues to work to pave a pathway for personalized treatments. The FDA recently released a draft guidance: https://answers.childrenshospital.org... on testing custom antisense oligonucleotides (ASOs) in patients.

credit: Adam Knee, Nancy Fliesler, Ryan Hartmann / Boston Childrens Hospital

Learn More: Bostonchildrens.org/milasen