The baby, KJ Muldoon of Clifton Heights, Penn., is one of 350 million people worldwide with rare diseases, most of which are ...

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific gene mutation, doctors say.

A Wigan family has teamed up with a national charity to help raise awareness of a rare genetic disease which affects both a ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

Another Breakthrough Summit West in the books, and what an excellent event! We kicked off the day with former FDA head Robert ...