A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy ...
Just days later doctors diagnosed him with a rare genetic disorder called Ornithine Transcarbamylase Deficiency (OTC). “I think the actual stats are about one in every 35,000 have a urea cycle ...
Ornithine transcarbamylase (OTC) deficiency is the most common urea cycle disorder. Urea cycle disorders are a group of inherited metabolic disorders that make it difficult for affected patients to ...
Steiner, MD Neonates with profound and prolonged hyperammonemia with coma due to urea cycle defect will have had ... N-acetylglutamate synthetase deficiency; OTC, ornithine transcarbamylase ...