By identifying the critical role the urea cycle plays in its development, his findings could pave the way for new medications to treat this currently incurable disease. In a study recently ...

Scientists have uncovered how ammonia wreaks havoc on liver cells by damaging mitochondria, leading to a dangerous cycle of toxicity. This discovery provides new hope as an existing drug, YAQ-005, ...

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.

For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that ...