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Today, we discuss the first gene therapy approval for Sanfilippo syndrome, learn of a rare gene variant linked to dramatically heightened lung cancer risk in non-smokers, and look ahead toward the confirmation hearing for the nominee to lead the FDA.
About the Sanfilipo approval: I first wrote about the effort to develop a therapy a decade ago. I get melancholy sometimes when I think of how many kids have declined or died before their families can get cutting-edge therapies approved for children who are not their own. But science takes time, and biotech can produce a better tomorrow, no?
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