Philadelphia-area family raises awarness about rare condition that prevents son from smiling

A Bucks County family is trying to raise awareness about a rare syndrome that has paralyzed their son's face. Chalfont, Bucks County, is lit up with purple lights for a brave little boy and the ...
The Manila Times

Crinetics Initiates Phase 2/3 Pediatric Trial Evaluating Atumelnant in Congenital Adrenal Hyperplasia (CAH)

First in class study of an investigational ACTH receptor antagonist aims to address significant unmet need in children and adolescents with classic CAH ...

New scoring system could more accurately predict shunt failures in children

Using information in medical records for more than 1,100 emergency room visits, researchers at Johns Hopkins Children's ...

FDA approves first treatment for Menkes disease in children

The U.S. Food and Drug Administration has approved the Zycubo (copper histidinate) injection as the first treatment for ...
The Chronicle-Journal

Going to Bat for Earlier Diagnoses in Cushing's Syndrome — Savannah's Story

As a college softball player, she was ready for any challenge on or off the field before symptoms of Cushing's syndrome, also ...