Urea cycle disorders (UCDs) are a group of diseases ... but not as much as they need. You may not spot symptoms for months or even years. A buildup of ammonia (you may hear the doctor call ...

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

These symptoms reflect ammonia’s dangerous ... Sometimes, it may also result in a coma or death. Urea cycle disorders: The urea cycle is a metabolic pathway in the liver that converts toxic ...

A baby in Pennsylvania has become the first recipient of an experimental genetic therapy that targets a rare urea cycle disorder. The treatment, which involves customizing the gene editing process ...

Details for the oral presentations are as follows: Title: Targeting Regulatory RNAs with Antisense Oligonucleotides for the Potential Treatment of Urea Cycle ... of-function disorders, of which ...

Zevra Therapeutics has partnered with the National Urea Cycle Disorders Foundation (NUCDF ... encephalopathy and with other neurological symptoms.” The website outlines a four-step testing ...