Rett syndrome (RTT) is a rare, severe, and progressive disorder that mostly affects girls of all races. It can impact your child’s ability to speak, walk, eat, or breathe easily. It was named after ...

A University of Virginia School of Medicine scientist is developing an innovative gene therapy she hopes will slow disease progression and improve movement, coordination and communication in children ...

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 ...

The US Food and Drug Administration has approved Acadia Pharmaceuticals’ Daybue Stix (trofinetide), a dye and preservative-free powder formulation of trofinetide, for oral solution, to treat Rett ...

With strong 1-year returns and upside potential, Taysha Gene Therapies, Inc. (NASDAQ:TSHA) secures a spot on our list of the ...

This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...

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 ...

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 ...

What is a Rett Syndrome? Rett syndrome is a rare but severe genetic neurological and developmental disorder that affects the development of brain. This disorder is only found in females. Girls ...

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 ...