Inside STAT: How doctors raced to stop a young girl's rare disease

DR. TIMOTHY YU, LEFT, WITH MILA AND HER MOTHER JULIA VITARELLO. (KATHERINE C. COHEN / BOSTON CHILDREN’S HOSPITAL)

Mila was just six years old when she was diagnosed with Batten's disease, a rare neurodegenerative disorder that is always fatal. But her parents, Julia Vitarello and Alek Makovec, now have a glimmer of real hope. With the help of scientists, regulators, and Mila’s family, an experimental therapy — developed in record time — now holds the potential to stop her disease in its tracks roughly a year after she was diagnosed. Her case serves as a proof-of-concept case study in the effort to rapidly develop and deliver precision therapies that are tailored to a particular patient. STAT's Meghana Keshavan has the story here.