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

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

Broad Institute via YouTube Direct link

From Rare Disease Parent to CEO: Gene Therapy for Dravet Syndrome

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4 of 8

From Rare Disease Parent to CEO: Gene Therapy for Dravet Syndrome

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

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

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