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

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.

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

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

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

A Southampton family needs your help to find a perfect match for their son, who is in need of a liver transplant. Last Friday, Rhett Powell turned one, a special milestone he celebrated with his ...

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 Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Rett syndrome epidemiology in the 7MM, i.e., the United States, EU5 ...

Rett syndrome is a rare genetic neurological condition that typically emerges in infancy and primarily affects girls, though occasional cases have been observed in boys. The disorder causes ...

Dublin, May 08, 2025 (GLOBE NEWSWIRE) -- The "Rett Syndrome - Pipeline Insight, 2025" clinical trials has been added to ResearchAndMarkets.com's offering.This report provides comprehensive insights ...

Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT), an organization working to cure Rett Syndrome, today ...

Rett syndrome is a rare developmental disorder, most often due to a mutation in the MECP2 gene on the X chromosome, but it’s not usually inherited. Advances in genetics have launched a new era ...