USAResearch and clinical trials
Experimental gene therapy in Rett syndrome: all of the first 12 patients gained at least one skill
An early result that has not yet been published in a peer-reviewed journal, or comes from a small, uncontrolled study. Treat it with caution.
New results were announced for an experimental gene therapy developed for Rett syndrome. The treatment is given once by injection into the spinal fluid. In the first part, all 12 patients aged 6–21 years gained at least one developmental milestone within 12 months. Among 29 patients in total, no serious side effects related to the treatment were reported. The data come from a company announcement, and the study has no comparison group.
What does it mean for patients and families?
The results have not yet been published in a peer-reviewed journal. The treatment is not approved; the first results of the main study are expected in 2027.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Rett syndrome
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.