Alice Melao,  —

StemoniX and Atomwise will work together to discover and develop small molecules that could be potential new therapies for people with Rett syndrome. This collaboration will take advantage of StemoniX’s human microOrgan platform and combines it with Atomwise’s artificial intelligence technology to expedite the development…

A Phase 3 clinical trial is recruiting girls and young women with Rett syndrome to explore the safety and efficacy of the investigational oral therapy trofinetide. The study, named LAVENDER, is planning to enroll 180 girls and women, 5 to 20 years old, at clinical sites in the…

Treadmill walking exercises can help improve gait speed and dynamics in people with Rett syndrome, a study suggests. However, the investigators cautioned that additional research is warranted to confirm these findings and explore the beneficial impact of treadmill training in this patient population. The study, “Kinematics associated with…

Several compounds already approved by the U.S. Food and Drug Administration (FDA) can increase the expression of the KCC2 protein in nerve cells, easing motor and respiratory symptoms in a mouse model of Rett syndrome. The study, “Pharmacological enhancement of KCC2 gene expression exerts therapeutic effects…

The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has endorsed Anavex 2-73 to receive orphan designation for the treatment of Rett syndrome, according to Anavex Life Sciences, the therapy’s developer. The proposal will now be evaluated by the European Commission, which will take into consideration…

Imbalance of thyroid hormones may contribute for the underlying biological mechanisms involved in Rett syndrome, results from a preclinical study suggest. The findings, “Altered Gene Expression of Thyroid Hormone Transporters and Deiodinases in iPS MeCP2-Knockout Cells-Derived Neurons,” were published in the journal Molecular Neurobiology. Rett syndrome…

Microswitches can be used as assistive technology to help improve choice-making and activity engagement in children with Rett syndrome, according to a recent study. Led by researchers in Italy, the study “Experimental Examination and Social Validation of a Microswitch Intervention to…

Dietary supplementation with the amino acid L-serine improved motor and communication skills in a young girl with atypical Rett syndrome caused by a genetic mutation in the GRIN2B gene, a case report shows. The patient’s positive outcomes suggest that this dietary therapeutic approach may be helpful for those with…

Preclinical data from mouse studies support initiating first-in-human clinical trials to explore the safety and effectiveness of the investigational gene therapy AVXS-201 in patients with Rett syndrome. Recent results have shown that one-time treatment with AVXS-201 is safe and well-tolerated in mice and non-human primates and supports the production…

A study in rats has provided new clues as to how the brain works to control body movements, which may have implications for disorders such as Rett syndrome. The study, “Strong neuron-to-body coupling implies weak neuron-to-neuron coupling in motor cortex,” was published in the journal Nature…