News

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…

The hands and feet of people with Rett syndrome have a lower skin temperature than people without the disease, correlating with impairments in the autonomic nervous system, a study reports. Skin temperature was measured using infrared thermal imaging (IRT), which uses infrared radiation, not visible to the human eye.

The global prevalence of Rett syndrome is estimated to be 7.1 cases per 100,000 female individuals, according to the first known meta-analysis ever done to estimate the number of girls and women worldwide with the rare neurodevelopmental disorder. This finding “may facilitate planning of therapeutic trials in this disease,…

Girls and women with Rett syndrome who participated in a three-month, at-home physical therapy program with occasional remote supervision met or exceeded most therapy goals and gained in motor abilities, a study reports. “The proposed activity programs … improved gross and fine motor skills, and enhanced the daily physical…