Long-term Phase 1/2 data suggest that Neurogene’s NGN-401 gene therapy may produce continuing developmental gains across several functional domains in Rett syndrome. The small, uncontrolled dataset remains preliminary, however, leaving the larger Embolden registrational trial to determine whether those improvements are reproducible, clinically persuasive and sufficient for regulatory approval.
Discover why Daybu’s CHMP turnaround could reshape Rett syndrome treatment in Europe and what clinical and access hurdles remain.
Find out how Taysha’s TSHA-102 Rett syndrome update could shape gene therapy development, BLA planning and future disease-modifying care.
Acadia Pharmaceuticals heads to AAN 2026 with Daybue, Nuplazid, and ACP-711 data. Read what the updates could mean for its neurology strategy.
Acadia Pharmaceuticals has launched DAYBUE STIX for Rett syndrome in the U.S. Read what this means for adoption, tolerability, and market strategy.
Acadia seeks EU re-examination after CHMP rejects trofinetide for Rett syndrome. Read the regulatory and clinical implications.
Acadia Pharmaceuticals has announced U.S. Food and Drug Administration approval for DAYBUE STIX (trofinetide) for oral solution, a new powder-based formulation of its existing drug DAYBUE used in the treatment of Rett syndrome. The approval covers use in adult and pediatric patients two years and older and is based on bioequivalence data, not new clinical