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.
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Parent-led funding campaigns to develop gene therapies for rare diseases are especially prevalent, and for good reason. Rather than finding a drug that can fill the void left by a protein lost to a single-gene disorder, gene therapy holds the promise of replacing the defective gene itself—of a cure. Just one of the thousands of single-gene disorders has an FDA-approved gene therapy, but through hard work and determination, some parents hope to change that.
Some crowdfunding campaigns have been astoundingly successful: they’ve raised millions of dollars to fund basic research and, later, clinical trials that have likely saved children’s lives. Donations can, however, only carry a therapy so far before a pharmaceutical company must grab the baton—an outcome that’s not guaranteed, even when a gene therapy shows promise in early clinical trials. And such therapies may not be able to turn back the clock on damage that’s already done, making cinematic happy endings to these stories unlikely.
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Yet for the families raising funds to combat rare diseases, any gain—whether in prolonging the life of one’s own child, sparing other families the same heartache, or some combination of the two—counts.
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