Sex-specific changes in the gut microbiome and associated metabolites came before disease progression in a mouse model of Rett syndrome, a study demonstrated. Notably, the study showed that female Rett mice models mimicked the syndrome’s microbial and molecular signatures better than male Rett mice, which are primarily used in…

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…

Acadia Pharmaceuticals and Stoke Therapeutics are teaming up to develop and commercialize new RNA-based therapies — using Stoke’s proprietary research platform, TANGO — for the potential treatment of Rett syndrome. As part of the collaboration, Stoke will receive a $60 million upfront payment from Acadia. “Stoke’s…

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…