Hope, news, and help for Rett families
We gather Rett syndrome news and explain it in plain words. Every story links to where it came from, so you can read more.
Latest News
This is not medical advice. Talk with your doctor.
Lab study backs the NGN-401 gene therapy
Neurogene makes a gene therapy called NGN-401. A new lab study found it raised the MeCP2 protein within one week, and the level stayed steady for months. MeCP2 is the protein that is missing or not working in Rett. The therapy is now in a late-stage trial called Embolden.
Source: Rett Syndrome News
Longer-term update from the NGN-401 early trial
Neurogene shared data from its early trial of 10 people. The company says every person gained at least one new skill, and no new serious side effects tied to the treatment were reported since October 2025. These are company-reported results and still need to be confirmed.
Source: Neurogene press release
Brain stimulation helped memory in Rett mice
Scientists at Baylor College of Medicine and Texas Children's studied deep brain stimulation in mice with Rett. It helped new brain cells grow and improved learning and memory in the mice. This was in mice, not people.
Source: Baylor College of Medicine