Applied Therapeutics’ rare disease drug has failed another late-stage clinical trial, adding to the woes of a candidate that ...

Govorestat failed to meet its primary endpoint in a Phase II/III trial for a rare form of Charcot-Marie-Tooth disease, a few ...

The outcome of the review has come as a major disappointment for patients and their families, with the Galactosemia Foundation calling the outcome "devastating" and urging the FDA to "reconsider ...

Acute liver failure (ALF) in children differs from that observed in adults in both the etiologic spectrum and the clinical picture. Children, particularly very young ones, do not demonstrate ...