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 ...

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.

Madison Sisson tells PEOPLE why she began sharing her journey raising two sons with leukocyte adhesion deficiency on TikTok.

Dead butt syndrome occurs when the gluteus medius stops firing due to unuse or sitting all day. Here are 5 exercises to help activate your glutes right now.

The biotech has received written agreement from the agency through the START pilot program on key aspects of its trial design for evaluating NGN-401.