Iqra Mumal, MSc,  —

Newron Pharmaceuticals is expanding the International Rett Syndrome Burden of Illness Survey to Europe and Australia in partnership with the worldwide Rett patient advocacy community, following strong participation from caregivers and medical professionals in the United States. Launched last November in the U.S., the survey is being extended…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to sarizotan for the treatment of breathing difficulties in people with Rett syndrome. This designation provides priority review to investigational treatments that have the potential to provide clinically meaningful benefits for people with…

Abnormal breakdown of lipid (fat) molecules known as sphingolipids may be involved in the development of Rett syndrome, and their levels could be used as a biomarker for the disorder, a pilot study reports. The research, “Sphingolipid Metabolism Perturbations in Rett Syndrome,” was published in the journal…

Researchers have discovered the genetic mechanisms involved in female inheritance and X chromosome silencing. This process, known as X chromosome inactivation, can potentially be manipulated for the treatment of disorders arising from defects in the X chromosome, including fragile X syndrome and Rett syndrome, a study reports. The…

A high-fat diet can lead to extreme obesity in Rett syndrome patients due to excessive calorie intake, and to brain networks unable to fully control feeding behavior, a mouse study suggests. The easy availability of high-fat foods in modern diets may further compound the problem — whose root cause is genetic…