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 developmental disorder that is the leading cause of severe intellectual disability in females. MECP2 mutations are the major genetic mutation associated with Rett syndrome.

Rett syndrome is a rare neurological and developmental disorder that leads to a gradual decline in motor skills and language.

In his postdoctoral work at Baylor College of Medicine and UVA, Bajikar and his team began studying how mutations in a gene called MECP2 contribute to the development of Rett syndrome. Their ...

Rett syndrome is a devastating neurological disorder caused mainly by mutations in the MECP2 gene found on the X chromosome. The disease causes deficits in brain function that lead to behavioural ...

(Nasdaq: ACAD) today announced that the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for trofinetide for the treatment of Rett syndrome ...

26(1), 1570–1580. 5 Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999; 23(2): 185-188.

3 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 In preclinical studies, deficiency in MeCP2 function is thought to lead to impairment in synaptic communication ...