STAT+: New gene therapy, to be priced at $4.25 million, has already transformed children’s lives
The world's most expensive treatment offers a near-cure for young children and redemption for its developer, but steep challenges still loom.
by Jason Mast and Megan Molteni
Mar 20, 2024
1 minute
Every morning, Victoria Rasberry gets up before 6 a.m., walks to the bedroom down the hall, and helps her 8-year-old daughter Addi slide into a set of contraptions
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