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Researchers are harnessing AI to pioneer a groundbreaking therapeutic approach for Rett Syndrome, offering new hope for improved diagnosis and personalized treatment.
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.
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.
Key to the discovery of vorinostat as a potential Rett syndrome therapy was the Wyss Institute's computational nemoCAD pipeline that enabled the team to predict drug candidates not based on a ...
The annual scientific congress on Rett syndrome, organized by the International Rett Syndrome Foundation and held recently in ...
Using an AI-driven drug discovery process, the drug known as vorinostat was identified as a promising treatment for Rett syndrome.
Stifel maintained its Buy rating with a target of $46, noting the competitive landscape in the Rett Gene Therapy sector and the significance of forthcoming data from Neurogene. 3rd party Ad.
More than thirty years since its 1993 founding, Catherine Owen Adams and Elizabeth Thompson—the R&D combo that has led Acadia ...