Marisa Wexler, MS, senior science writer —

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…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Early, intensive training in motor and memory skills before symptoms develop may delay disease progression and help children with Rett syndrome retain specific milestones, a study in mice suggests. Observed benefits were not evident when training was given to symptomatic animals, its researchers wrote, supporting “genetic screening of newborns…

A fine balance of proteins called cohesins is important for the normal inactivation of the X chromosome, new research suggests. Better understanding how the activity of the X chromosome activity is regulated could be important for treating conditions like Rett syndrome, which is caused by a mutation in a…

Researchers have identified a set of RNA molecules that are dysregulated in Rett syndrome and may be relevant biomarkers for the disorder. The study, “Unraveling Molecular Pathways Altered in MeCP2-Related Syndromes, in the Search for New Potential Avenues for Therapy,” was published in the journal…

Novartis Gene Therapies is planning to submit an investigational new drug application (IND) for OAV201, its Rett syndrome gene therapy candidate, by the end of the year, the company announced in a letter to the Rett community. If the IND is approved by the U.S. Food and Drug Administration…