Next Generation Solutions for Rare Disease Patients

Explore cutting-edge approaches to rare disease research and treatment through this symposium session featuring three expert presentations on next-generation solutions for rare disease patients. Learn about the role of sequencing, registries, and patient partnerships in clinical laboratories with Niall Lennon from Broad Clinical Labs, who discusses how these tools are advancing rare disease diagnosis and research. Discover innovative gene delivery methods through Arya Rao's presentation on harnessing cell-type specific gene regulation for targeted therapeutic approaches. Examine dystrophin-based treatments and their potential universal applications with Michelle Rengarajan's research on making dystrophin therapies accessible to all patients. Gain insights into scalable technologies and biological advances that are driving progress toward treatments and cures for patients with rare genetic diseases, presented as part of the Ladders to Cures Accelerator Symposium hosted by the Broad Institute.