The girl, now 16 months, began receiving the critical protein her body can't make while still in the womb. The treatment was delivered through a needle inserted through the mother’s abdomen and guided into a vein in the umbilical cord. The disease killed two of the girl's sisters before they turned 3.
Using a protocol developed at UCSF, physicians have successfully treated a fetus with a devastating genetic disorder for the first time, and the child is now thriving as a toddler.