BENGALURU: Awareness about rare diseases can sometimes prevent serious conditions from being overlooked. One such condition ...

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 ...

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.

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

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.

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.

B rain parasites could potentially be used to help treat cognitive disorders like Alzheimer’s Disease and Parkinson’s, ...

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

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 ...