The US Food and Drug Administration has approved Acadia Pharmaceuticals’ Daybue Stix (trofinetide), a dye and ...

ROB: RETT SYNDROME IS A RARE DISEASE THAT AFFECTS 1 IN 10,000 GIRLS. IT’S EVEN MORE RARE IN BOYS. A RETT SYNDROME DIAGNOSIS IS LIFE-CHANGING FOR FAMILIES. SAMANTHA BRANT, WHOSE DAUGHTER MACY LIVES ...

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which ...

Daybue Stix, a strawberry-flavored trofinetide powder mixable with water-based liquids, gains FDA approval for Rett syndrome treatment.

ST. LOUIS, Mo. (Ivanhoe Newswire) – Imagine your toddler learning to walk and talk and then all of a sudden, your child stops and starts regressing inside of themselves. That’s what happens to ...

An expert discusses the current treatment landscape of Rett syndrome including the goals of treatment and implications for utilization management strategies with the disease. David Lieberman, MD, PhD: ...

What is Rett syndrome and why does it affect only girls? Rett syndrome is a rare neurological disorder that causes victims to regress intellectually, gradually robbing their ability to think and move.

The son of NBC News chief correspondent Richard Engel, Henry Engel died eight months ago at age 6 — but he's still at the center of the research that will hopefully prevent other children from losing ...

An expert explains how Rett syndrome is diagnosed, how commonly it is misdiagnosed, disease progression, and which providers are best positioned to manage a Rett Syndrome patient. David Lieberman, MD, ...

In commemoration of Rett syndrome awareness month this October, Acadia Pharmaceuticals is launching an awareness campaign to shine a light on patients' journeys and show that each person living with ...

New Haven-based Biohaven Pharmaceutical Holding Co. Ltd. said its investigational compound has been granted orphan drug designation by the U.S. Food and Drug Administration to treat patients with Rett ...