There are a number of exciting therapeutic strategies being pursued that attack the root cause of MEPC2 Duplication Syndrome. These include antisense oligonucleotides therapy, small interfering RNA oligonucleotides therapy, and genome editing. Cures however are not developed in isolation. They require a myriad of resources that encourage and enable progress.

Dr. David Pehlivan and Dr. Bernhard Suter will provide an update of clinical studies currently being pursued including a clinical severity scale for MDS, a patient registry and biomarker efforts.

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The Rett Syndrome Research Trust is singularly focused on a cure for Rett syndrome and related MECP2 disorders. Achieving success takes a deep understanding of the relevant science, an environment built for scientific collaboration, and experience with drug and business development. Today we are building on incredible scientific and industry momentum by implementing six genetic-based strategies that tackle Rett from every angle. Every single company pursuing a cure for Rett is able to do so because they leveraged discoveries and resources that were generated with RSRT funding. RSRT is the home of the Rett cure community, and we are confident that together we will cure Rett syndrome.