Marisa Wexler, MS,  senior science writer—

The Rett Syndrome Research Trust (RSRT) has received a $1 million donation to help develop a cure for Rett syndrome. The gift, provided by Alba Tull, philanthropist and co-founder of Tull Family Foundation (TFF), will support the RSRT’s CURE 360 initiative, a project that aims to be…

Novartis announced it is scrapping plans to move OAV201, an investigational gene therapy for Rett syndrome, into clinical trials. “The totality of the pre-clinical data [on OAV201] does not support a path forward to human clinical trials,” according to the company’s third quarter report for this year.

The International Rett Syndrome Foundation (IRSF), an organization focused on accelerating research to treat and cure Rett syndrome, has been appointed to the National Health Council (NHC) Standard of Excellence program. “This is a great honor. It recognizes IRSF’s leadership in advancing treatments and cures for patients…

Abnormally slow or shallow breathing in Rett syndrome develops in part due to altered inhibitory signals in the brain, research done in a rat model of the disease suggests. Data also indicate that therapeutic approaches that amplify the brain’s inhibitory signals might be useful for Rett-related breathing problems. These…

Participating in school virtually — using a computer with a webcam — is socially and cognitively engaging for people with Rett syndrome, according to a study in Italy. This finding “supports the idea that the children with RTT [Rett syndrome] can benefit from the use of technology-aided programs, such…

Acadia Pharmaceuticals is launching an open-label clinical trial of its oral investigational medication trofinetide in toddlers and young girls with Rett syndrome, the company announced in a letter to the Rett community. The 12-week Phase 2/3 trial, called DAFFODIL (NCT04988867), aims to enroll about 10 children, ages 2…

Acadia Pharmaceuticals has completed enrollment in the Phase 3 clinical trial LAVENDER, which is testing the investigational medication trofinetide as a treatment for Rett syndrome. Top-line results from the study are expected later this year, according to a press release. LAVENDER (NCT04181723) enrolled approximately…

The Ontario Rett Syndrome Association (ORSA) announced the awarding of two grants to support research into Rett syndrome in Canada. According to an ORSA press release, grants totaling CA$78,750 (about $62,800) will be given to two selected projects. The funding comes from The Hope Fund, which ORSA…

A music-based intervention helped to normalize certain social behaviors in a mouse model of Rett syndrome, researchers reported. Findings also suggested that repeated, regular exposure to music altered the activity of certain genes in particular brain regions, yielding clues as to the biologic mechanisms through which the intervention works. The…

Alcyone Therapeutics, a new company focused on developing gene therapies for disorders that affect the nervous system, announced its official launch. One of its lead candidates, ACTX-101, is an investigational gene therapy for Rett syndrome now in preclinical testing with a goal of bringing into clinical trials in patients…