Karen Wright worked her way
through the small gathering
outside her 4-month-old
daughter’s room in pediatric
intensive care. It seemed to her as
if everyone at UF Health Shands
Children’s Hospital had come to
witness this historic moment.
Wright, nervous but thrilled, told a nurse, “I think I’m
going to cry.”
Near her, a pharmacist delicately held a clear bag
containing a plastic syringe. Syringes deliver drugs. But to
Wright, this one would do so much more. It would give
her daughter, Londyn, the hope of a full, happy life. The
pharmacist handed the bag to a nurse who took it into
the room, where cartoons danced on a TV screen.
Wright leaned down and whispered to her baby, “This
drug is going to save your life.”
In June, Londyn became the first baby in the country
treated with a newly approved gene therapy for a rare
and debilitating neuromuscular disease since it won
federal approval in May. UF Health doctors intravenously
infused the girl with Zolgensma during an hourlong
procedure to treat her spinal muscular atrophy, or SMA,
a rare and deadly disorder that affects the ability to walk,
eat and breathe. Untreated, most babies die before
reaching their first birthday.
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BY DOUG BENNETT AND BILL LEVESQUE