
The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas.
KJ Muldoon is walking and getting ready to celebrate the holiday season at home with his parents and three siblings.
KJ was born last year with a genetic disorder called carbamoyl-phosphate synthetase 1 deficiency. Also known as CPS1 deficiency, the metabolic condition affects only 1 in 1.3 million babies and often leads to life-threatening outcomes.
When he was 6 months old, doctors began giving KJ a groundbreaking new treatment -- a personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia.
Doctors save baby's life with first-ever gene fix for deadly rare disease
The gene-editing therapy works like a "molecular spell-checker,” finding and fixing the specific genetic error causing his condition.
After three infusions and after spending the first 10 months of his life in a hospital, KJ was discharged and sent home in June.
Baby saved by gene-editing therapy 'graduates' from hospital, goes home
Since then, KJ has continued to grow and thrive with his family, celebrating his first birthday at home and taking part in one of the family’s favorite rituals -- cheering for their beloved Philadelphia Eagles.
Doctors hope new technology like CRISPR gene-editing therapies can be the key to treating more than 7,000 rare diseases, such as sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy, that affect approximately 30 million people across the country.
LATEST POSTS
- 1
A Sweet Choice: Pick Your #1 Cake! - 2
Dominating Monetary Administration: A Bit by bit Manual for Making an Individual Financial plan - 3
FACT FOCUS: Trump sows confusion on number of childhood vaccinations - 4
The architect of Iran’s military survival remains defiant - 5
Traveling Alone: An Excursion of Self-Disclosure
Best Exciting ride: Which One Rushes You the Most?
Spanish police and soldiers track boars, reinforce farm security amid swine fever outbreak
Cyber Monday 2025: Save over 70% on HBO Max with this Prime Video streaming deal
We may be witnessing the messy death of a star in real time
Alice Wong, founder of the Disability Visibility Project, dies at 51
Sean 'Diddy' Combs faces new sexual assault allegations, currently under investigation by Los Angeles Sheriff's Department
Muslim nations condemn new Israeli death penalty law
Which One Energizes You the Most These Tech Developments
RFK Jr. wants to scrutinize the vaccine schedule – but its safety record is already decades long












