Forging a New Path for Medicine
from RARECast
Amy Dockser Marcus, in her new book We the Scientists, tells the story of a group of parents of children diagnosed with the rare and fatal genetic lysosomal storage disorder Niemann-Pick disease type C. When they were confronted with the fact that no treatment existed or would likely be developed in time to save the lives of their kids, they began collaborating with themselves, researchers, and physicians to accelerate the path to a treatment. We spoke Marcus about the lessons learned from the experience of the Niemann-Pick disease type C community, how these parents took an active role in the drug development process as citizen scientists, and how their efforts reflect a broader change in the way biomedical research is conducted.
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What this episode covers
Amy Dockser Marcus, Pulitzer Prize-winning journalist and author of the new book We the Scientist, discusses how parents of children with the rare lysosomal storage disorder Niemann-Pick disease type C took an active role in the drug development process as citizen scientists, lessons learned from their experience, and how their efforts reflect a broader change in the way biomedical research is conducted.
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