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 ...

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

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 ...

(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 ...