Rett syndrome is associated with a complex phenotype that is unique in every aspect of its presentation, clinical physiology, chemistry, and pathology. Years of concentrated observations have ...

The neurodevelopmental disorder Rett syndrome involves mutations in the transcriptional repressor MECP2. Two groups now show a role for MECP2 in postmitotic mouse neurons. You have full access to ...

More information: Osman Sharifi et al, Sex-specific single cell-level transcriptomic signatures of Rett syndrome disease ...

As exemplified by Rett syndrome, for ASD there is no discernible phenotype in the first year of life. Presymptomatic molecular diagnosis can offer an opportunity to initiate treatment early to ...

NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

Phase 2a TREE study assesses safety and efficacy of leriglitazone Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone ...

Decision on orphan drug designation for NTI164 to treat Rett syndrome expected in ‘due course’ US Food and Drug Administration granted orphan designation for NTI164 in Rett syndrome in ...

TRUMBULL, Conn., March 13, 2025 (GLOBE NEWSWIRE) -- The Rett Syndrome Research Trust (RSRT), the organization working to cure Rett syndrome, is pleased to announce that Amy Gilliland, President of ...

(RTTNews) - Acadia Pharmaceuticals Inc. (ACAD) announced the publication of clinical data from the open-label DAFFODIL study, which evaluated the long-term safety and efficacy of DAYBUE or ...