Accelerating Rare Disease Research: Patients as Partners - Rare Disease Day 2023

Explore a comprehensive conference talk on accelerating rare disease research through patient partnerships, presented by the Broad Institute of MIT and Harvard for Rare Disease Day 2023. Delve into topics such as mitochondrial diseases, ultra-rare genetic neurodevelopmental disorders, gene therapy for Dravet syndrome, and inherited retinal degeneration. Learn about initiatives leveraging patient power to advance research, including the Rare as One project. Gain insights from expert speakers and a panel discussion on empowering progress through patient collaboration. Discover the Broad Institute's plans for a rare disease initiative, and understand the crucial role of patient foundations in developing therapies for rare conditions.

Welcome Remarks
Spotlight on Mitochondrial Diseases
Working Hand in Hand with Patient Foundations to Develop Therapies for Ultra-Rare Genetic Neurodevelopmental Disorders
From Rare Disease Parent to CEO: Gene Therapy for Dravet Syndrome
Rare as One: Leveraging the Power of Patients to Accelerate Disease Research
Inherited Retinal Degeneration: Therapeutic Landscape
A Panel Discussion: Partnering with Patients to Empower Progress
Plans for a Rare Disease Initiative at Broad