STAT Plus: After a bespoke therapy rescues a young girl, the FDA considers advance of individualized treatments
Back in 2016, a 6-year-old named Mila was diagnosed with Batten disease, a progressive and incurable genetic syndrome that would brutally strip away her sight and her ability to walk, and would cause dozens of seizures each day. The condition is fatal.
But her parents, Julia Vitarello and Alek Makovec, refused to lose hope. They contacted Dr. Tim Yu, a neurologist at Boston Children’s Hospital, who discovered the precise genetic mutation that caused her disease — and was able to devise an experimental treatment designed exclusively for Mila. His work is one of the first examples of therapies created for the benefit of a single patient — an “N of 1” study.
