Neurogene’s Rett syndrome gene therapy has preliminary data supporting safety and efficacy of the one-time treatment. But a late-breaking report of a serious complication in a patient who ...

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

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.

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.

Multiple clinical trials in gene therapy for Rett syndrome are now underway, all possible because of RSRT-funded research.

Rett syndrome treatment typically includes physical therapy, occupational therapy, speech and language therapy, nutritional support, and medications.

NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT ...