At the International Rett Syndrome Foundation’s (IRSF) conference earlier this month in Minnesota, which I attended, there was quite a lot of talk in the scientific presentations about gene replacement, gene activation, gene silencing, and such. Also at the conference, there were quite a few young kids with Rett…
Discussion
All 10 patients with Rett syndrome treated with Neurogene‘s gene therapy NGN-401 continued to make developmental gains over 2.5 years of follow-up, with no plateau or loss of skills observed, according to long-term data from a Phase 1/2 clinical trial. The data showed that these gains deepened over time…
Discussion
The last time I went to the national conference devoted to Rett syndrome, my daughter Abby’s disability, I brought a bathing suit and a beach towel and finished the conference 10 pounds heavier than I had started it. I’m on Day 1 of the 2026 conference as I…
Discussion
Daybu (trofinetide), marketed as Daybue and Daybue Stix in the U.S., could become the first treatment for Rett syndrome neurobehavioral issues in the European Union, if the European Commission approves a committee’s recommendation. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive…
Rett syndrome is a rare genetic disorder that mostly affects females. The disease is characterized by normal early development, followed by slowed growth and a loss of motor control. Learn more about the disease here.
Most cases are caused by a mutation in the MECP2 gene. Scientists have identified mutations in CDKL5 and FOXG1 genes in those who have atypical or congenital Rett. Learn more about what causes the disease here.
Rett syndrome is associated with problems in cognitive, sensory, emotional, and motor skills, and across the nervous system that controls involuntary (or autonomic) functions. Learn more about the symptoms of the disease here.
Get regular updates to your inbox.
