In the first 48 h after K.J. Muldoon’s birth last year, doctors noticed signs of lethargy and respiratory distress ... CPS1 deficiency is a urea cycle disorder in which a person lacks a liver ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

Fargo’s Aldevron helped create a custom gene-editing therapy that cured a baby of urea cycle disorder, a rare and potentially ...

A newborn boy, KJ, was diagnosed at birth with a rare and potentially fatal genetic disease. He received a novel, on-demand CRISPR therapy in record time.