After their apparent success treating a young girl with a drug tailored to counteract a genetic mutation that had given her a usually fatal brain disease, the researchers behind the innovative strategy have this week laid out criteria for similarly helping more sick children. But the U.S. Food and Drug Administration (FDA) is cautioning that such one-off therapies need to be thoroughly considered before moving ahead and carefully evaluated, in part because desperate parents sometimes perceive improvements from a treatment that are not real.
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