News

Children’s Hospital Los Angeles Expands Space for Rett Care

The Rett Syndrome Clinic at Children’s Hospital Los Angeles has been relaunched in an expanded space and expects to enhance individualized care for children with the disorder. The new Neurological Institute Outpatient Center opened this year at the hospital’s Sunset Boulevard campus. It aims to provide comprehensive care for children…

Brain Waves Captured by EEG Indicate Cognitive Therapy Benefits

Alterations in brain waves, measured using an electroencephalogram (EEG), revealed benefits of cognitive stimulation in Rett syndrome patients, a small study found. The benefits of cognitive stimulation were greater in younger girls, “highlighting that the younger the therapy is started, the better is the outcome,” the scientists wrote. The…

‘Encouraging’ Results Seen in Patients in LAVENDER Phase 3 Trial

Trofinetide, Acadia Pharmaceuticals’ experimental therapy, significantly reduced neurobehavioral symptoms and improved communication in girls and young women with Rett syndrome, according to top-line data from the Phase 3 LAVENDER clinical trial. These results — meeting the trial’s main and key secondary goals — were consistent across age ranges…

Problems in Neuronal Activation, Complexity Seen in Rett Model

Neurons activated at the same time — a state called neuronal synchrony — and with lesser complexity in the relationships between nerve cells of the brain and the body may contribute to the aimless and repetitive movements of Rett syndrome, a recent animal study suggests. The study, “Collapse of…

Rett Syndrome Research Trust Receives $1M Donation

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…

Continuous BDNF Supply by Stem Cells May Have Therapeutic Potential

Stem cells engineered to produce a constant level of a downstream target of the MECP2 protein, called brain-derived neurotrophic factor (BDNF), increased the development of new neurons and prevented body weight loss in a mouse model of Rett syndrome. The study with that finding, “Brain-Derived Neurotrophic Factor Secreting…