For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as ...

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...

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 baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

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

But his daughter, who was diagnosed with an ultra-rare genetic disorder in 2019, has lost the ability to speak. “She screams and giggles and cries and makes noises and sounds, but she doesn't ...

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

In a Brazilian village, a rare disease has affected many children. Research links it to cousin marriages, revealing how ...

At age 9, Mansour was diagnosed with a rare genetic disorder called NARS1. She will be the first patient to receive an experimental antisense oligonucleotide gene therapy to treat NARS1 symptoms.

Here is all you need to know about Lafora disease: Lafora disease is a rare and serious genetic disorder that affects the brain and nervous system. It belongs to a group of conditions known as ...

The 9-year-old girl was diagnosed with an ultra-rare genetic disorder that caused her to flatline once and has taken away her ability to speak “I'm devoting the rest of my life to this,” the ...

Most children of cousin couples are healthy, but the risk of rare genetic disorders increases when both parents carry the same mutation. “If a couple is unrelated, the chance of having a child ...