Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
Science Daily: Pharmacology News
MAY 15, 2025
The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment. The process, from diagnosis to treatment, took only six months and marks the first time the technology has been successfully deployed to treat a human patient.
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