Ultragenyx posts late-stage trial win for gene therapy in urea cycle disorder

Ultragenyx Pharmaceutical (RARE) announced on Thursday that its gene therapy candidate DTX301 reached the main goal in a late-stage trial for ornithine transcarbamylase (OTC) deficiency, the most ...
JSTOR Daily

Ornithine-Urea Cycle Enzymes in the African Lungfish, Protopterus aethiopicus

The presence of all five enzymes of the ornithine-urea cycle has been demonstrated in the liver of the African lungfish Protopterus aethiopicus. Levels of activity of the rate-limiting enzymes, ...
MPR

Gene Therapy DTX301 Reduces Ammonia in Patients With OTC Deficiency

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.
GEN

Memory Loss in Alzheimer’s Disease Pivots on Urea Metabolism

Targeted inhibition of an enzyme linked to the urea cycle called ornithine decarboxylase 1 (ODC1) in star-shaped cells in the brain called astrocytes, could prove beneficial in restoring memory ...
Seeking Alpha

iECURE's therapy GTP-506 for urea cycle disorder gets orphan drug status in EU

The European Commission (EC) granted orphan designation to iECURE's lead product GTP-506 to treat Ornithine Transcarbamylase (OTC) deficiency. OTC deficiency is a type of urea cycle disorder. It is a ...