Baby saved by gene-editing therapy 'graduates' from hospital, goes home

ABC ^ | 06 04 2025 | Sony Salzman

[yesthatjallen]

KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week.

KJ has spent the majority of his life at Children’s Hospital of Philadelphia after being diagnosed with a one-in-a-million, deadly genetic disease shortly after birth. Working quickly, his doctors were able to use new gene-editing technology called CRISPR, designing a bespoke treatment just for him.

The treatment, first infused into his body at seven months old, seems to have worked. KJ’s body, which was fighting a toxic buildup of ammonia, began to thrive and he quickly gained weight appropriate for a baby of his age.

Wearing a cap and gown to symbolize his “graduation” from the hospital, baby KJ was discharged home to his parents and siblings on Tuesday after spending 307 days at Children’s Hospital of Philadelphia. Doctors and nurses gathered for a "clap out" on his way out the hospital doors, and he was escorted home by local law enforcement.

KJ's metabolic condition, called carbamoyl-phosphate synthetase 1 deficiency, affects about 1 in 1.3 million people. The disease kills 50% of babies by early infancy.

SNIP

[Ronaldus Magnus III]

Rumor has it that Planned Parenthood is not only protesting this baby surviving, they are also Judge shopping for a retroactive abortion.

[TheDon]

CRISPR is an amazing technology. KJ was born a girl! 😉. Good bless KJ!

[GaltAdonis]

Children’s Hospital of Philadelphia is a great place.
It’s been a number of years since I told this story on this board, so I’ll do it again.
Friends of mine had a son. After several days they went home from the hospital.
All was well. Until the baby in his crib turned blue, and was barely breathing.
They called an ambulance, then off to a local hospital, then onto a helicopter
to rush him to CHOP. Bottom line is that he had a congenital birth defect,
a whole in his heart. He almost died. They operated and fixed it.
He is over 30 now, and is a college graduate and a mechanical engineer.
6 foot 3 and healthy as a horse.

God bless the talented great folks at CHOP!

[Revel]

This is all fine until they use the technology for other purposes that have nothing to do with saving a life or adverse medical condition. And they will.

[stonehouse01]

Wonderful story! Our daughter had a life saving bone marrow transplant at CHOP in 2014 and she is now just starting her second year of residency there this month!!! She received her M.D. from Thomas Jefferson Sidney Kimmel Medical school in Philly last year. Can you believe it? She helped take care of this little guy for a short time while she was on that unit.

CHOP is awesome.

[GaltAdonis]

Yes they are!