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Meta-Analysis of Genetic Studies Essential to Discover New Therapeutic Targets for Rett Syndrome, Reviewers Contend

Implementation of meta-analysis — a statistical approach that combines information from different studies — using data from studies on the protein and genetic landscape of Rett syndrome is essential to discover new therapeutic targets for this disease, according to a recent review. The study, “Genome-wide transcriptomic and proteomic…

European Agency Recommends Orphan Designation for Anavex 2-73

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…

Thyroid Hormone Imbalance May Contribute to Rett Syndrome, Study Suggests

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…