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Inherited Retinal Degeneration: Therapeutic Landscape
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Accelerating Rare Disease Research: Patients as Partners - Rare Disease Day 2023
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- 1 Welcome Remarks
- 2 Spotlight on Mitochondrial Diseases
- 3 Working Hand in Hand with Patient Foundations to Develop Therapies for Ultra-Rare Genetic Neurodevelopmental Disorders
- 4 From Rare Disease Parent to CEO: Gene Therapy for Dravet Syndrome
- 5 Rare as One: Leveraging the Power of Patients to Accelerate Disease Research
- 6 Inherited Retinal Degeneration: Therapeutic Landscape
- 7 A Panel Discussion: Partnering with Patients to Empower Progress
- 8 Plans for a Rare Disease Initiative at Broad
