KJ Muldoon was born with a rare genetic disorder called CPS1 deficiency. That is a rare genetic disorder that affects the body’s ability to remove excess nitrogen from the blood.
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Half of all babies with his condition die within days. But his parents chose to pursue treatment rather than comfort care.
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Doctors at the Children’s Hospital of Philadelphia worked with top scientists and companies to create a special gene therapy just for KJ. They used a tool called CRISPR to fix the exact genetic mistake that caused his illness.
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After three doses, KJ got much better. He can now eat normal amounts of protein, needs less medicine, and has stayed stable even through illnesses that could have been very dangerous before.
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It’s still too soon to know if he’ll need a liver transplant, but for now, he’s doing well and getting ready to go home.
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h/t: NY Times
Photos: Children’s Hospital of Philadelphia
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