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Baby leaves CHOP after 307 days thanks to gene-editing therapy

After more than 10 months at Children’s Hospital of Philadelphia, the infant who received a pioneering gene-editing therapy went home. KJ Muldoon risked irreparable brain damage from his condition, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, in which his liver could not process protein. CPS1 deficiency is deadly in more than half of cases. Muldoon's parents took him home Tuesday after 307 days at the hospital, CHOP announced.
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