A UC San Diego lab recently announced a pair of scientific breakthroughs for Pitt-Hopkins and Rett syndromes that are slated ...

An expert provides an overview of the RSBQ and CGI score measurement tools and other tools/methods of assessment used in clinical practice. David Lieberman, MD, PhD: There are a few outcome measures, ...

Rare genetic disorder shuts off motor skills; mental acuity may still be there. May 9, 2012— -- For the first year of life, Chelsea Coenraads was a perfectly normal child, playing peek-a-boo and ...

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.

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 ...

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: ...

One little boy’s unique form of a debilitating genetic disorder may help researchers find a treatment for him and others. By studying the unusual form of Rett syndrome in the 2-year-old son of NBC ...

Most cases of Rett syndrome are caused by a genetic mutation that affects a gene called MECP2 present on the X chromosome. This gene codes for the MeCP2 protein, which is essential for the normal ...

The Rett Syndrome Research Trust (RSRT) is delighted to announce that Timothy A. Riley, Ph.D. has joined the organization in the role of Chief Scientific Officer and Chief Business Officer. Randall ...

One mother’s mission to find a cure for her daughter’s debilitating brain disorder may be paying off. Scientists now say that a cure for Rett Syndrome, which strikes mostly girls, may be just around ...

The son of NBC News chief foreign 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 ...