New Drug Designed for Only One Patient

DENVER (AP) – A 9-year-old Boulder girl with a neurological disease is being treated with a one-of-a-kind drug designed specifically for her.

It’s the first time the U.S. Food and Drug Administration has approved a drug for a single person, and it’s an example of what the future might hold for precision medicine.

The drug is called milasen, after Mila Makovec. The 22-letter genome sequence in the drug’s recipe matches the sequence in one of Mila cells that is broken.

It may not be enough to save Mila, because it can only slow the process of cell degeneration and not replace the cells in her brain that have already died.

But her doctors say the case has advanced specialized drug development in a way that could open new paths for children with incurable genetic diseases.