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…
News
All 12 girls and women with Rett syndrome given the gene therapy candidate TSHA-102 in a clinical trial gained or regained at least one developmental milestone within one year, with consistent responses seen across differing ages and disease severity levels, new trial data show. Longer-term follow‑up data from Part…
All 25 participants in the Phase 3 Embolden study have received their doses of NGN-401, Neurogene’s experimental gene therapy for Rett syndrome, surpassing the trial’s initial enrollment target ahead of schedule. “We are pleased to have completed dosing in the Embolden trial of NGN-401 within our original timeline while…
Stimulating the vagus nerve, part of the autonomic nervous system that regulates involuntary functions, paired with sound tones, may support the development of future therapies for communication deficits in Rett syndrome. Researchers found that VNS-tone pairing changed protein levels in the brain’s sound-processing region in a rat model of…
Mutations in the MECP2 gene  — the main cause of Rett syndrome — may disrupt early development by altering how DNA is three-dimensionally organized within chromatin, the complex of DNA and proteins that packages genetic material inside cells, according to a study in China. “This work provides a new…
Abnormally high activity of the MECP2 gene can profoundly disrupt immature brain cells during early development, but appears to have only modest effects in mature nerve cells, according to a new study. The findings may have important implications for the development of gene therapies for Rett syndrome, which is…
Families of girls and young women with Rett syndrome reported reduced overall quality of life, with emotional well-being emerging as the most affected area, according to a study from Brazil. Better family quality of life was associated with higher income, private health insurance, regular school attendance, and the absence…
Unravel Biosciences announced it has begun dosing in a proof-of-concept clinical trial evaluating its experimental therapy, RVL-001, in people with Rett syndrome. Richard Novak, PhD, Unravel’s co-founder and CEO, said this marks “an important milestone for our company and for [people with] Rett syndrome” in a company…
Blocking the signaling of leptin — a hormone involved in appetite regulation, motor function, and behavior — eased some Rett syndrome-like symptoms in a mouse model of the disease, a study found. Data showed that reducing leptin signaling either pharmacologically or genetically in male mice with Rett-like symptoms helped…
In mouse models of Rett syndrome, disease-related changes in the MeCP2 protein were linked to widespread disruptions in histones, proteins that help package DNA and regulate gene activity within cells. That’s according to the study, “Spatially resolved mapping of histones reveals selective neuronal response in Rett syndrome,” published…
Recent Posts
- The International Rett Syndrome Foundation conference, then and now
- EU panel backs Daybu to treat Rett syndrome behavioral issues
- The day my daughter with Rett syndrome became invisible
- Finding the space to breathe after years of Rett syndrome caregiving
- All Rett patients in trial achieve motor milestones with gene therapy
- A doctor’s retirement, and those stairs we climbed for Rett syndrome
- Embolden gene therapy trial finishes dosing ahead of schedule
- Rett syndrome makes my kid a different sort of baseball fan
- Hindsight illuminates the early signs that my daughter had Rett syndrome
- VNS-tone pairing alters brain proteins in Rett syndrome rat model