Rett syndrome is a devastating rare genetic childhood disorder primarily affecting girls. Merely 1 out of 10,000 girls are ...

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 ...

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares. Last week, the company reported that NGN-401 had shown ...

The New York biotech's update focused on data from four patients treated with a low dose of Neurogene's NGN-401 therapy in female Rett syndrome patients, which it said showed "meaningful gains of ...