Taysha Gene Therapies has unfurled more data in the developmental disorder Rett syndrome, linking its gene therapy to improvements in the first adults and children to receive low doses of the ...

Real World Data from LOTUS Study Evaluating Long-term Efficacy and Tolerability Outcomes of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Developmental Medicine and Child Neurology ...

In the comfort of her Miami home, 34-year-old Julianne Espinosa lies in bed captivated by videos of Disney princesses flashing across her television screen. As echoes of “The Little Mermaid” fill her ...

Scientists investigating the severe developmental disorder known as Rett syndrome have discovered a series of crucial molecular changes that occur long before symptoms appear. The findings could be ...

Texas Children's Hospital, an internationally-recognized, top-ranked children's hospital and pediatric research center affiliated with Baylor College of Medicine, is the first to deliver a novel gene ...

Anavex released data from the EXCELLENCE trial, designed to evaluate ANAVEX 2-73 in 92 children aged 5-17 with Rett syndrome. The trial had two co-primary endpoints, Rett Syndrome Behaviour ...

The My Rett Ally web app, powered by mejo, is a new resource to support caregivers of individuals with Rett syndrome. My Rett Ally, a web application powered by mejo, is specifically designed for Rett ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has granted marketing authorization of DAYBUE™ (trofinetide) for the treatment of Rett ...

Children’s of Alabama and UAB have announced the creation, upon University of Alabama System Board of Trustees’ approval, of the Sarah Katherine Bateh Endowed Professorship in Rett Syndrome as a ...

(Mass Appeal) – It’s a rare and cruel disorder with symptoms that can begin as early as 6-months old–Rett Syndrome. Children with Rett Syndrome require round the clock care for life. Rett Syndrome ...

On an unseasonably warm day in late February, scientists at Kunming Medical University’s Experimental Animal Center in the far southwestern province of Yunnan injected a female crab-eating macaque ...