The Food and Drug Administration has approved expanded use of leucovorin calcium tablets for treating cerebral folate ...

The FDA declined to endorse leucovorin as a treatment for autism, but approved the drug to treat cerebral folate deficiency, ...

Rian McCann is almost seven years old. Her parents, Danielle and Frank described her as kind, sassy, smart and above all, resilient. Rian was born with CDKL5 Deficiency Disorder — which prevents her ...

The Food and Drug Administration approved leucovorin to treat a neurological disorder with some symptoms similar to autism.

On Thursday, Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused ...

By Ahmed Aboulenein and Julie Steenhuysen WASHINGTON, March 10 (Reuters) - The U.S. FDA on Tuesday approved leucovorin, a decades-old generic drug, for use against an ultra-rare disorder called ...

The European Commission has granted marketing authorisation in the European Union (EU) for Ztalmy ® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with ...

Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat. Topline data from ACTIVATE-Kids study of mitapivat in children who are not ...

ATLANTA, Dec. 8, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today presented positive efficacy and safety results from the GEMZ phase 3 study investigating adjunctive fenfluramine ...

Vitamin A deficiency should be considered in children with unexplained vision loss, particularly those with restrictive eating disorders such as avoidant/restrictive food intake disorder. Early ...