Author Archives: Marisa Wexler MS

Acadia Opening Phase 2/3 Trial of Trofinetide in Young Girls, 2-5

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…

LAVENDER Trial Enrollment Complete

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…

ORSA Grants Support 2 Rett Research Projects in Canada

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…

Music Therapy Seen to Make Mice in Rett Model More Sociable

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.

Skills Training Given Before Symptoms Evident May Help Rett Children

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…