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Sanfilippo
Families of Children with Rare Diseases Fuel Gene Therapy Research
The Scientist Staff | May 1, 2018 | 10+ min read
Crowdfunding can power investigations into diseases that would otherwise receive little attention.
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
Diana Kwon | May 1, 2018 | 5 min read
When the O’Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.
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