Treatments & Trials
This is not medical advice. Talk with your doctor.
trofinetide (Daybue)
In 2023 the FDA approved trofinetide (Daybue) as the first treatment for Rett syndrome. It is for adults and children 2 years and older. It is a liquid taken by mouth or through a G-tube, twice a day.
The most common side effects were diarrhea and vomiting. The label says to stop laxatives before starting and to call your doctor if diarrhea happens.
Source: FDA
Source: DailyMed drug label
Taysha: TSHA-102 gene therapy
TSHA-102 is a one-time gene therapy given by spinal injection. Taysha finished dosing 17 patients in its REVEAL trial and 4 young children in its ASPIRE trial. It expects results and FDA feedback in the first half of 2027. It is not approved yet.
Source: Taysha Gene Therapies
Neurogene: NGN-401 gene therapy
NGN-401 is a one-time gene therapy. Neurogene finished dosing 25 patients in its Embolden trial. It is not approved yet.
Source: Rett Syndrome News
Source: Neurogene
Find a clinical trial
ClinicalTrials.gov is a free government list of research studies. You can search for "Rett syndrome" and your state. Ask your doctor if a study might be a good fit.
The IRSF also keeps a list of Rett treatments being studied.
Source: IRSF Research Pipeline