Archive for precision medicine
Against all odds: Mila’s unique mutation, and her own custom drug
As a baby and toddler, Mila was healthy, active, and — in some ways — even advanced for her age. She began speaking before she turned 1, went skiing for the first time at age 2, and was a fearless climber on furniture and rock walls. However, after she turned 3, Mila developed a strange ... Read More
Shooting for the moon: From diagnosis to custom drug, in one year
One weekend in January 2017, Timothy Yu, MD, PhD, was relaxing at home when his wife said, “Hey Tim, will you look at this post on Facebook? This might be something you’d be interested in.” Doctors had just diagnosed a young girl named Mila in Colorado with Batten disease, a rare and fatal neurodegenerative disorder. ... Read More
Small samples, big data: A systems-biology look at a newborn’s first week of life
The first week of a baby’s life is a time of rapid biological change. The newborn must adapt to living outside the womb, suddenly exposed to new bacteria and viruses. Yet scientists know surprisingly little about these early changes. Reporting in Nature Communications, an international research team provides the most detailed accounting to date of the ... Read More