When six-year-old Mila Makovec was diagnosed with a devastating neurological disorder called Batten disease, the happy, active girl had already lost her ability to see, to say most words, and to walk without help. There was no known treatment for her fatal condition.
Then, in a dramatic demonstration of the power of personalized medicine, doctors in Boston created a treatment just for her. In only eight months, they found the genetic cause of Mila’s disease, designed a drug to overcome the error, and began giving it to Mila via an injection in her spine, in what’s believed to be the first individually tailored treatment of its kind.