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

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Rare Disease Day 2023
Accelerating Rare Disease Research: Patients as Partners
Broad Institute of MIT and Harvard

Chapters
00:00 Welcome Remarks
Anna Greka and Belinda Termeer
10:39 Spotlight on Mitochondrial Diseases
Vamsi Mootha
45:06 Working Hand in Hand with Patient Foundations to Develop Therapies for Ultra-Rare Genetic Neurodevelopmental Disorders
Yael Weiss
01:02:47 From Rare Disease Parent to CEO: Gene Therapy for Dravet Syndrome
Daniel Fischer
01:15:48 Rare as One: Leveraging the Power of Patients to Accelerate Disease Research
Tania Simoncelli
01:34:15 Inherited Retinal Degeneration: Therapeutic Landscape
Eric Pierce
01:51:11 A Panel Discussion: Partnering with Patients to Empower Progress
Moderator: Samantha Baxter
Panelists: Charlene Son Rigby, Eric Pierce, Daniel Fischer, Yael Weiss, Vamsi Mootha, Tania Simoncelli
02:37:12 Plans for a Rare Disease Initiative at Broad
Anna Greka

Copyright Broad Institute, 2023. All rights reserved.
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