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

Doctors will have to monitor the boy for years to ensure the experimental treatment is effective long-term. But for now, it's a godsend.

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

PHILADELPHIA (WPVI) -- An infant, who was born with a deadly disease, is now thriving, thanks to a medical first performed at ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as ...

Sandra A. Banta-Wright, MN, RNC, NNP; Robert D. Steiner, MD Neonates with profound and prolonged hyperammonemia with coma due to urea cycle defect will have had a neurological insult to the brain ...

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.

It is driven by the increasing prevalence of UCDs. The global urea cycle disorder (UCD) treatment market is projected to grow by $215.4m from 2024 to 2028, at a CAGR of 3.2%, according to Technavio.

My long standing focus has been the study of human inborn errors of metabolism and structural birth defects of the skeleton. We have applied genetic approaches to the study of biochemical genetic ...

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific ...