June 24, 2025

Tag: urea cycle disorder

Baby KJ gene therapy sparks hope at CHOP

Baby KJ gene therapy sparks hope at CHOP

A Baby’s Battle from Birth Baby KJ gene therapy began after he was born with a potentially fatal genetic disorder on August 1, 2024. He received his diagnosis in a flash. He had inherited a severe urea cycle disease, namely CPS1 deficiency, according to the doctors. Because of the elevated ammonia levels in his blood, […]

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