World's first patient treated with CRISPR gene editing therapy at CHOP returns home
A ten-month-old baby who has been at the Children's Hospital of Philadelphia (CHOP) since birth finally got to go home with his family.
KJ Muldoon made headlines around the globe a few weeks ago for being the first person in the world to receive a breakthrough gene editing therapy that is customized to the patient.
The family said after KJ received three infusions in February, March and April. Doctors said the results are very promising so far.
CLIFTON HEIGHTS, Pa. - An emotional homecoming occurred in Delaware County for a ten-month-old baby named KJ Muldoon who made headlines around the globe just a few weeks ago.
The backstory
KJ was born on August 1 and diagnosed with a rare metabolic disease called CPS1 that causes ammonia to build up to a toxic level in the body.
In February, doctors treated the infant with a breakthrough and historic CRISPR gene editing therapy, making KJ the very first patient in the world to receive this kind of personalized treatment.
KJ received additional infusions of the experimental therapy in March and April.
Doctors have told his parents the results so far are very promising.
What's New
"We went through all of the emotions. You're excited, you're nervous, but we're just glad that he's finally able to be home with us," said Nicole Muldoon, KJ's mother. "We've been operating like five plus one for so long and we're excited to be the six of us moving forward."
"We're trying to meet all his developmental milestones and kind of see what he's capable of, but he's already shown us how special he is and I think we're in for a treat."
KJ's family and friends hung up welcome home signs and colorful balloons as they waited in anticipation for his homecoming in Clifton Heights.
"It's just been a really long fight for him," said Dee Aaron, KJ's grandmother. "Miracles do happen, and it really is a miracle. We didn't think he'd be here."
"We're so happy you're home big guy," said Cathy Franklin, KJ's great-grandmother. "He's beautiful, and his parents have been remarkable just remarkable. Stayed so strong and we just prayed for ten months. Here he is!"
The staff at CHOP dressed KJ up in a graduation cap and gown for his send-off, and they sent him out the hospital doors in great numbers, cheering on his health and recovery.
Once the family made it out of the hospital, police from Upper Darby and Radnor Township escorted the family from CHOP all the way to Clifton Heights. The nonprofit The Delco Group helped arrange the special police escort.
"We made one phone call yesterday with Ken Piree from Radnor right to Upper Darby Township. It was just within seconds and that's what you get in Delaware County. Everybody gets each other's back here," said John Port of The Delco Group.
What you can do
The community has also raised tens of thousands of dollars in a gofundme campaign which will now help support KJ's medical needs moving forward.
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