Marisa Wexler, MS,  senior science writer—

A recent meal or snack does not affect the pharmacological profile of trofinetide, an experimental oral treatment for Rett syndrome, according to findings from a Phase 1 study in healthy adults. Food can affect how a drug moves through a patient’s body, or its pharmacological profile, the researchers…

Unravel Biosciences, which is working toward a first clinical trial to test its investigational therapy RVL001 in people with Rett syndrome, announced that it has partnered with the rare disease health platform TMA Precision Health to identify eligible patients for recruitment into the study.

Treatment with a modified version of the MeCP2 protein, one designed to better get into cells, markedly extended lifespans in a mouse model of Rett syndrome, a study reports. The MeCP2 protein here was modified by adding a sequence derived from the human immunodeficiency virus (HIV) — a virus…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

The protein MeCP2 normally forms a complex with several other proteins in the brain that helps to regulate the development of connections between brain cells, a study reported. These findings offer new insight into how Rett syndrome and other conditions caused by mutations affecting MECP2 can develop. The study…

A molecular sensor of infections called the NLRP3 inflammasome is overactive in immune cells of people with Rett syndrome, a new study reveals. The study, “The constitutive activation of TLR4-IRAK1- NFκB axis is involved in the early NLRP3 inflammasome response in peripheral blood mononuclear cells of…

The Rett Syndrome Research Trust (RSRT) has invested $3.1 million in a slew of research projects as part of its CURE 360 initiative. CURE 360, which launched last year, aims to help move preclinical advances toward treatments for Rett syndrome — with the ultimate goal of developing…

A cellular signaling pathway called the JNK pathway is overactive in cell and animal models of Rett syndrome, and blocking it lessens signs of the disease in these models, according to a new study. “These results suggest that JNK inhibition could offer an attractive therapeutic strategy to tackle RTT…

The MECP2 gene — mutations in which cause most cases of Rett syndrome — may be important for encoding early childhood traumas into the genetic wiring of the brain, affecting behaviors, a study in mice suggests. Titled “MeCP2 haplodeficiency and early-life stress interaction on anxiety-like behavior in adolescent…

A newly found mutation in the MECP2 gene that causes Rett syndrome was described in a recent report. The report highlights some of the considerations that need to be taken into account when testing for an unknown Rett-causing mutation. Here, a testing glitch showed an artificial result from…