Gene therapy offered this 7-year-old freedom. The price: a grueling year
STAT
DECEMBER 13, 2023
PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just after her sixth birthday. She lost her appetite and was often doubled over in pain. She continued getting blood transfusions but her doctors struggled to manage side effects that threatened her organs. By the time she turned 7, the doctors told her parents they had to do something — soon.
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