February 2026 Neurogene Inc. announced that the U.S. FDA granted Breakthrough Therapy Designation to NGN-401, its investigational gene therapy for Rett syndrome, supporting the accelerated development of the program.
December 2025 Unravel Biosciences received approval from Colombia's INVIMA to initiate the RVL-001 clinical study for Rett syndrome, expanding its clinical development program into Latin America.
June 2025 Neurocores announced that the European Medicines Agency (EMA) granted Orphan Drug Designation to KIT-14 for the treatment of Rett syndrome, supporting the development of its rare disease pipeline.