In mouse models of Rett syndrome, disease-related changes in the MeCP2 protein were linked to widespread disruptions in histones,…
Articles by Marisa Wexler, MS
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…
The Embolden clinical trial, a registrational study testing Neurogene‘s gene therapy NGN-401 in people with Rett syndrome, has…
After expatriation of a partnership agreement with Astellas, Taysha Gene Therapies regained full rights to TSHA-102, its experimental gene…
A gene therapy showing potential to restore lost developmental milestones in people living with Rett syndrome has earned breakthrough…