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.