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Families of Children with Rare Diseases Fuel Gene Therapy Research

External Link - Source: The Scientist

It’s a compelling narrative: A parent learns that his or her child has a fatal disease with no cure, and, though not a scientist, embarks on a quest to find some treatment. Such stories have played out in the plotlines of films such as Lorenzo’s Oil and Extraordinary Measures, on national morning shows and local news segments, and on crowdfunding pages to drum up support for the cause.

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