
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
The most effective method to Settle on Informed Conclusions about Senior Insuranc. - 2
Malaysia To Revive Search for Missing Flight MH370 - 3
Exploring Being a parent: A Survey of \Bits of knowledge and Guidance for Guardians\ Nurturing Book - 4
Figure out How to Stay away from Normal Handshaking Missteps - 5
The Secret Destinations Amex Says Will Be More Popular Than Bali by 2026
Ukraine proved this drone-killer works. Now, the West is giving it a shot.
Seven deaths possibly linked to malfunctioning glucose monitors
Vote In favor of Your Number one Kind Of Food Conveyance Administration
RFK Jr. says he's following 'gold standard' science. Here's what to know
Senior's Manual for Obtaining a Hyundai Ioniq EV: Tips
Minneapolis ICE shooting live updates: Protests continue over agent's killing of Renee Nicole Good; Walz puts National Guard on standby
Trump declares Christmas Eve and Dec. 26 federal holidays: What does that mean?
25 Most Beautiful Villages in France You Can Actually Visit
BioMarin to acquire Amicus Therapeutics for $4.8 billion in rare disease bet












