
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
Manual for Famous Beverages 2024 - 2
The most effective method to Shield Your Gold Ventures: Procedures and Precautionary measures - 3
Figure out How to Utilize Your Web based Advertising Degree to Break into the Tech Business - 4
Baikonur launch pad damaged after Russian Soyuz launch to International Space Station - 5
Island Travel Guide: Must-Visit Objections for 2024
Vote in favor of Your #1 BWM Vehicles
Real time features for Films and Programs
Exposure to neighborhood violence leads some Denver teens to use tobacco and alcohol earlier, new study shows
5 Chiefs That Changed Our Opinion on Film
'All Her Fault' ending explained: The shocking conclusion to the psychological thriller inspired by true events
Manual for Tracking down One of a kind Store Inns
Treason trial of South Sudan's suspended VP is further eroding peace deal, UN experts say
7 Powerful Techniques to Boost Efficiency with Your Cell Phone: A Thorough Aide
Unraveling the Specialty of Picking Your Ideal Travel Objective













