Scientists have uncovered how mutations in the MeCP2 gene lead to Rett syndrome, a devastating neurological disorder. By ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year ...

New research brings scientists one step closer to understanding Rett Syndrome, a rare genetic disease that affects 1 in ...

Researchers at the Josep Carreras Leukaemia Research Institute revealed a new aspect of Rett Syndrome’s pathogenesis that can ...

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 ...

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage biotechnology company focused on advancing adeno-associated ...

Taysha Gene Therapies' TSHA-102 tackles Rett syndrome’s root cause with its miniMECP2 transgene and scAAV delivery. So far, this is TSHA’s main value driver, and it appears safe and effective ...

When symptoms such as "noticing significant developmental delays in mobility, language, and social interaction since age two" are entered into a dialogue box, an AI large model can generate alerts ...

Discover Stoke Therapeutics' innovative RNA-based treatments like zorevunersen for Dravet syndrome, with a promising pipeline ...