Taysha Gene Therapies offers hope for Rett syndrome with innovative treatment, strong trial results, and promising regulatory ...

Rett syndrome is a neurodevelopmental disorder that primarily affects girls, most of whom have mutations in the transcription regulatory gene MECP2. However, mutations in MECP2 also have been ...

Rett syndrome is a rare neurological and developmental disorder that leads to a gradual decline in motor skills and language. An X-linked genetic disease, Rett syndrome occurs in approximately 1 in ...

In March 2023, the US Food and Drug Administration approved Acadia's Daybue (trofinetide) for treating Rett syndrome in adults and paediatric patients aged two years and above. The therapy is also ...

It's not easy to deal with tragedy, but for Colin Endres and the D-Y/CCT/CCA boys hockey team, they're honoring his sister ...

Rizzo is diagnosed with Rett syndrome, which leads to a regression of basic skills that a small child may have already learned. Her parents say that the volunteers at Sebastian Riding Associates ...

If granted marketing authorization, trofinetide will be the first and only approved therapy for Rett syndrome in the European Union (EU). Trofinetide is already approved in the United States and ...

Neuren Pharmaceuticals has announced it has received its share of the net proceeds from the sale of the Rare Pediatric ...