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 ...

High levels of ammonia kill liver cells by damaging the mitochondria that power the cells. But this can be prevented using an existing drug due to start clinical trials, finds a new study in mice led ...

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