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A gene therapy showing potential to restore lost developmental milestones in people living with Rett syndrome has earned breakthrough therapy status from the U.S. Food and Drug Administration (FDA). The experimental therapy, TSHA-102, is being developed by Taysha Gene Therapies. A breakthrough therapy designation…

October is Rett Syndrome Awareness Month, and supporters are going purple to bring attention to the rare genetic condition. “There’s no one right way to raise awareness,” the International Rett Syndrome Foundation (IRSF) says on its website. “Whether you’re posting on social media, sharing your story, or just starting…

Mutations in the MECP2 gene — which typically underlie Rett syndrome — cause problems in how neurons, or nerve cells, produce energy and communicate, and affect how well they can repair breaks to DNA. That’s according to the findings of a new study, done in both human brain cells…

One year of daily Daybue (trofinetide) treatment led to improvements in behaviors such as nonverbal communication, alertness, and social interaction and connectedness among children and adults with Rett syndrome. That’s according to published interim data from Acadia Pharmaceuticals‘ Phase 4 LOTUS study that assessed the safety and…

Using an oral cannabidiol (CBD) solution as an add-on treatment to antiseizure medications was shown to lower the frequency of drug-resistant seizures by more than half in about two-thirds of children with Rett syndrome in a study conducted in Italy. “Our findings suggest a strong trend toward the efficacy…

Girls with Rett syndrome often face challenges when it comes to eating and drinking, according to a 20-year analysis. Due to limited hand function and difficulties with chewing and swallowing, many are unable to feed themselves or handle a wide variety of foods and liquids. As these…

Reduced production of orexin and its receptors is associated with problems in sleep-wake cycles in a mouse model of Rett syndrome (RTT), a study suggests. Orexin is a signaling molecule that regulates sleep and wakefulness in the brain. While circadian rhythms that regulate the sleep-wake cycle were generally unaffected in…

Blocking a small RNA molecule that helps control whether certain genes are switched on or off reactivated the healthy copy of the MECP2 gene in a mouse model of Rett syndrome, increasing lifespan and activity, and improving breathing. While blocking miR‑106a appears to be “a feasible therapeutic strategy,” before…

Vorinostat, an approved lymphoma treatment sold as Zolinza, was found to ease both neurological and non-neurological symptoms in a mouse model of Rett syndrome in a new study. When an oral formulation of vorinostat was administered after symptom onset, the drug was even more effective than Daybue (trofinetide),…

Cannabidivarin (CBDV), a non-psychoactive compound found in cannabis, preserves cognitive function and improves motor coordination in a female mouse model of Rett syndrome, a study suggests. “These findings provide new insights into the role of CBDV in [Rett] and support for future research, highlighting its potential as a ……