In 2018, at age 7, Mila Makovec became the world’s first person to be treated with a drug made just for her. Languishing from Batten disease, a rare, fatal neurodegenerative disorder, she received an antisense oligonucleotide (ASO) drug designed to silence her genetic mutation, injected into the fluid around her spine through a lumbar puncture.
A team led by Timothy Yu, MD, PhD, in the Division of Genetics and Genomics and Manton Center for Orphan Disease Research at Boston Children’s Hospital, created the drug within the space of a year — a first in medicine. It was dubbed milasen.
