We the Scientists: How Patients Are Driving Research: 2023 RARE Advocacy Summit Keynote

October 12, 2023

We the Scientists: How Patients Are Driving Research and Why It’s Necessary for Finding Cures was the opening keynote for the RARE Advocacy Summit in San Diego California, September 19-21, 2023.

Hear how a daring team of doctors and parents of children diagnosed with the fatal genetic lysosomal storage disorder Niemann-Pick disease type C forged a new path for medicine. Amy Dockser Marcus is a Pulitzer Prize–winning reporter who followed patients, families, caregivers, and patient advocates and wrote a moving narrative of these patient advocates who are revolutionizing the way medical research is conducted. Her book, We the Scientists: How a Daring Team of Parents and Doctors Forged a New Path for Medicine, was published in February 2023.

Amy Dockser Marcus
Pulitzer Prize-winning Wall Street Journal Health and Science Reporter

Nicole Boice
Founder and Chief Mission Officer, Global Genes

Chris Hempel
RARE Mom & Advocate



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