Stimulating the vagus nerve — a large nerve that helps control unconscious bodily functions — partly normalized some measures of…
Articles by Marisa Wexler, MS
Abnormally high activity of the MECP2 gene can profoundly disrupt immature brain cells during early development, but appears to have…
Unravel Biosciences announced it has begun dosing in a proof-of-concept clinical trial evaluating its experimental therapy, RVL-001, in people…
In mouse models of Rett syndrome, disease-related changes in the MeCP2 protein were linked to widespread disruptions in histones,…
Different mutations that cause Rett syndrome can have divergent effects on brain cell activity, a study found. The findings…
Daybue Stix, a powder formulation of Daybue (trofinetide), is now widely available for eligible people with Rett syndrome…
Acadia Pharmaceuticals has hit a roadblock in its quest for European approval of trofinetide, the company’s oral therapy for…
A Phase 3 clinical trial testing the experimental gene therapy NGN-401 for Rett syndrome is underway and continuing to…
The pivotal REVEAL clinical trial — a Phase 3 study testing Rett syndrome gene therapy candidate TSHA-102 — has…
Note: This story was updated Dec. 5, 2025, to correct the primary outcome in the Embolden study. NGN-401, an experimental…