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…
News
Using a comparative genomics approach, researchers have identified three potential therapies for Rett syndrome that are being used or tested for other indications. “This study highlights the potential of comparative genomics to accelerate drug discovery, and yields potential new avenues for the treatment of [Rett],” the researchers wrote. …
Brain organoids grown from stem cells derived from people with Rett syndrome reproduced patterns of electrical brain activity that resembled seizures, a hallmark of the condition, a study demonstrated. Use of anti-seizure medications restored electrical activity to normal levels, supporting the use of brain organoid models to investigate underlying…
Treatment with fluoxetine, a type of antidepressant sold under the brand name Prozac, increased the number of cells producing the protein MeCP2 — defective in most cases of Rett syndrome — in several brain regions of a mouse model of the disease, a study reported. These effects were…
The lack of a working MeCP2 protein — the most common cause of Rett syndrome — prematurely closes a window of neuronal plasticity in a brain region involved in social memory, a study showed. Neuronal plasticity is the brain’s ability to re-wire itself, to strengthen or weaken its neuronal connections, in response…
The loss of MECP2 protein — defective in most cases of Rett syndrome — in the lungs may result in breathing problems, according to a mouse study. The findings suggest that this may due to altered amounts of fats in the lungs, and could have important implications for treating…
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…
A first-generation university student has been awarded a six-year, $466,669 grant from the National Institutes of Health (NIH) to fund his doctoral and postdoctoral research into brain-derived neurotrophic factor (BDNF), a protein that plays a role in Rett syndrome. The Virginia Tech student, Raymundo Hernandez, called the NIH grant…
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