Gene therapy offered this 7-year-old freedom. The price: a grueling year
For a 7-year-old girl, a gene therapy for beta thalassemia offered freedom. The price: a grueling year.
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.
Several months later, Shelby laid in her mother’s arms on a hospital bed, napping. Her medical team — who deemed the late June 2023 day Shelby’s “re-birthday” — gathered around, preparing to administer a life-changing treatment. Her doctor, Tim Olson, connected the first of two syringes to a small tube that led to a port in Shelby’s chest. Both syringes were filled with a translucent, slightly beige liquid. Everyone in the room, holding their breaths, knew it was worth more than if it were liquid gold: $2.8 million, to be exact.
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