Taysha Gene Therapies Is Showing Promise For Rett Syndrome
Taysha Gene Therapies offers hope for Rett syndrome with innovative treatment, strong trial results, and promising regulatory ...
Medscape21 天
Recent Progress in Rett Syndrome and MECP2 Dysfunction
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.
Pharmabiz23 天
Acadia Pharma seeks European marketing approval for trofinetide for the treatment of Rett ...
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 ...
AFP24 天
Acadia Pharmaceuticals Submits Marketing Authorization Application to the European ...
(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 ...
capitalbrief25 天
Neuren shares dive despite EU regulatory progress
More news: Neuren Pharmaceuticals was the worst performing ASX 200 company in early trading after its US partner Acadia Pharmaceuticals applied for approval to market its Rett syndrome treatment, ...
Pharmaceutical Technology25 天
Acadia submits MAA to EMA for Rett syndrome therapy
Trofinetide is intended to treat Rett syndrome, a neurodevelopmental disorder. Credit: SewCreamStudio/Shutterstock. Acadia Pharmaceuticals has submitted a marketing ...