More than thirty years since its 1993 founding, Catherine Owen Adams and Elizabeth Thompson—the R&D combo that has led Acadia ...

Researchers from Radboud University Medical Center and King's College London present a comprehensive framework for validating ...

The annual scientific congress on Rett syndrome, organized by the International Rett Syndrome Foundation and held recently in ...

Researchers from Universidade de Lisboa and the Gulbenkian Institute of Molecular Medicine have found that chronic ...

Researchers are harnessing AI to pioneer a groundbreaking therapeutic approach for Rett Syndrome, offering new hope for improved diagnosis and personalized treatment.

Using an AI-driven drug discovery process, the drug known as vorinostat was identified as a promising treatment for Rett syndrome.

Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are born with it and much fewer boys. It is caused by mutations in the MeCP2 gene ...

An AI-enabled drug discovery approach identified a potentially game-changing treatment for Rett syndrome, which has been advanced from the lab bench to an FDA Orphan Drug Designation in record time.

For more information, visit www.neurogene.com. About NGN-401 NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome.