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

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

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

The pivotal trial for Neurogene's Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that ...

The purple and pony-themed ducks benefit Izzie's Duck Drive for a Cure for Rett syndrome. Izzie Boom is a 15-year-old Concord High School student teaching other students and teachers about the ...

Rett syndrome is a rare genetic neurological disorder that occurs most often in girls (1 in 10,000 births), more rarely in boys, and leads to severe impairments, affecting nearly every aspect of life.

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced details of ...

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 ...

At 12 weeks, trofinetide-treated patients had a 5.1-point reduction in the Rett Syndrome Behaviour Questionnaire (RSBQ) scale, compared to a 1.7-point drop for those one placebo.

Currently, there is no treatment or cure for the disease, so a few years ago Rhett’s family decided to hold a 5K to raise awareness and funds for research.