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Biopharma Tool Kit for Rett Syndrome Development Programs

July 10, 2021

The goal of the Rett Syndrome Research Trust (RSRT) is to advance the most promising curative approaches that take aim at the root cause of the disease - MECP2 mutations. We identify therapeutic strategies and incubate programs with the objective to transition them to biopharma. We have developed a variety of resources, the Biopharma Tool Kit, to support biopharma development programs. This webinar provides a brief overview of the Biopharma Tool Kit including new non-clinical models, advanced MECP2 imaging and quantification technology, patient biorepository, up-and-coming clinical development tools including a digital natural history study, the Rett Syndrome Global Registry, the development of digital endpoints and the first communication-focused caregiver-reported outcome measure.

Whether you are pursuing a Rett program or still exploring, we invite you to watch this webinar and encourage you to share this information with colleagues who may be interested.

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RSRT Clinical Initiatives

RSRT Scientific Initiatives