News

The first patient has been dosed with TSHA-102, an investigational gene therapy by Taysha Gene Therapies, to treat Rett syndrome. The Phase 1/2 trial, dubbed REVEAL (NCT05606614), is still recruiting up to 12 women with Rett syndrome at its single site in Montreal, Canada. Early safety data…

Levels of anxiety and depression in girls and women with Rett syndrome are influenced both by the individual’s type of MeCP2 gene mutation — the underlying cause of the neurodevelopmental disorder — and the presence of sleep problems, a new study reports. In particular, patients carrying p.Arg294*, a mutation…

TSHA-102, an experimental gene therapy by Taysha Gene Therapies, can increase the levels of MeCP2 — a protein that’s not found in sufficient amounts or is dysfunctional in people with Rett syndrome — while causing no harm to cells that have normal levels of the protein. That’s according…

The Phase 1/2 REVEAL study, which is testing the investigational gene therapy TSHA-102 in women with Rett syndrome, is expected to dose its first participant in the next month or two, according to Taysha Gene Therapies, its developer. “Screening is completed, and dosing is now scheduled for our first…

The Assistance Fund (TAF), an independent patient assistance organization that helps patients and families with out-of-pocket healthcare costs, has expanded its program to include people with Rett syndrome and their families. The fund seeks to help with expenses such as health insurance premiums and copays, therapy administration costs,…

Daybue (trofinetide) is easing the neurobehavioral symptoms of Rett syndrome with acceptable safety in girls ages 2 to 4, according to early data from the open-label DAFFODIL Phase 2/3 trial. These results were consistent with previous clinical trials in Rett patients starting at age 5 that supported…

Individuals with Rett syndrome experience a “substantial” clinical and economic burden throughout their life, according to a new real-world study in the U.S. Altogether, the study found, the high prevalence of clinical symptoms, increased use of healthcare resources, considerable healthcare costs, and great reliance on medications and supportive…

Daybue (trofinetide) is now available in the U.S. to treat Rett syndrome in patients 2 and older, according to the therapy’s developer Acadia Pharmaceuticals. The announcement comes about a month after Daybue became the first therapy to be approved for Rett syndrome by the U.S.

The recent U.S. approval of Daybue (trofinetide) as the first treatment for adults and children with Rett syndrome represents a game changer, offering hope for the care of this patient community, say researchers, advocates, and caregivers. “The availability of a drug that helps with symptoms caused by Rett…

Mice in Rett syndrome models showed a loss of brain volume before disease symptoms were evident, with brain shrinkage worsening over time, an MRI study showed. Delays in brain growth were observed in these mice, and brain growth stopped and appeared to reverse in older female Rett mice. Because…