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

What is Cushing's syndrome? Cushing's syndrome is a hormonal disorder. It’s caused when you have high levels of the hormone cortisol over a long time. Cushing's syndrome is fairly rare. It most often ...

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

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.

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

(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 ...

The news: Neuren Pharmaceuticals' Rett syndrome treatment, trofinetide, is a step closer to receiving regulatory approval in the European Union, after its US partner Acadia Pharmaceuticals submitted a ...

Neuren’s partner Acadia Pharmaceuticals submits marketing authorisation application in Europe for trofinetide for treatment of Rett syndrome Compumedics achieves a record $32.8m in sales orders in H1 ...