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 expert discusses the current treatment landscape of Rett syndrome including the goals of treatment and implications for utilization management strategies with the disease. EP: 1.

There is already 1 company, Taysha Gene Therapy, that launched a trial in women [aged 18 and older] with Rett syndrome in Canada. I don’t know if anyone’s been dosed yet, but at least that is ...

A major breakthrough in Rett syndrome research has been achieved through the combination of AI-driven drug discovery and innovative disease modeling. This approach led to the identification of ...

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 ...

Neurogene Announces Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome. Share. Written agreement from FDA for single-arm, open-label, ...

Insights into neurons that cause symptoms of Rett syndrome could guide new therapy search. ScienceDaily. Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2016 / 06 / 160628114618.htm.

T he Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a genetic disease mostly affecting girls that causes severe neurologic impairments, robbing them of the ...

Dublin, Feb. 28, 2024 (GLOBE NEWSWIRE) -- The "Rett Syndrome - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering.This report provides comprehensive ...

Answer: Rett syndrome is a neurodevelopmental disorder in which a child develops along a typical path until about 6-18 months of age. After this time, there is a gradual loss of a broad range of ...

Bird says there are three likely approaches to a Rett syndrome treatment. One is a gene therapy that would give people a functional MECP2 gene. That's going to be especially tricky, as too much of ...