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: ...

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 ...

Biomed Industries Presents Promising Phase 2/3 Clinical Results of NA-921 for Rett Syndrome at Orphan Drug Summit 2026 ...

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 late June 2026, Acadia Pharmaceuticals reported that the EMA’s Committee for Medicinal Products for Human Use issued a ...

A new UC Davis MIND Institute study offers critical insights into Rett syndrome, a rare genetic condition that affects mostly girls. The research reveals how this condition affects males and females ...

Gene therapy may offer hope for people with a rare childhood disease called Rett syndrome. Around one in 10,000 girls are born each year with Rett syndrome, a rare genetic condition that impacts brain ...

The European Medicines Agency (EMA) has recommended granting marketing authorization to Daybu (trofinetide, Acadia Pharmaceuticals B.V.) to treat neurobehavioral symptoms of Rett syndrome in adults ...

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 ...

Acadia stock jumps 7% as CHMP backs Daybu for EU approval in Rett syndrome, with the European Commission set to make a final decision in the coming months.

SAN FRANCISCO--(BUSINESS WIRE)--Herophilus announced today that in vivo studies underway of its lead candidate HRP-12975 are funded in part by the Rett Syndrome Research Trust (RSRT). HRP-12975 is the ...

Daybue was evaluated in a randomized, double-blind, placebo-controlled, 12-week study. Researchers at the University of Alabama at Birmingham enrolled 12 study subjects in the multi-site trial.