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The Robert G. Fenley Writing Awards: Basic Science Staff Writing - Silver

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Infusion of Hope UF Health patient first to receive new gene therapy for neuromuscular disease since FDA approval
Infusion of Hope UF Health patient first to receive new gene therapy for neuromuscular disease since FDA approval

“UF Health Patient First to Receive New Gene Therapy for Neuromuscular Disease since FDA Approval” by Doug Bennett and Bill Levesque
University of Florida Health

Little Baby Londyn was diagnosed with a rare and debilitating neuromuscular disease not long after her birth called spinal muscular atrophy. It’s a deadly disorder affecting a child’s ability to walk, eat and breathe. Untreated, most babies die before their first birthday.

Londyn, just 4 months old, became the first baby in the nation to be infused with a potentially life-saving drug called Zolgensma at University of Florida Health.

It works by using a small, harmless virus to deliver functional copies of a gene that is mutated or missing in SMA patients. A dysfunctional gene, known as survival motor neuron 1, causes nerve cells to malfunction and die. That leads to chronic and often fatal muscle weakness.

UF Health researchers have done pioneering work in the field of gene therapy.

Londyn’s mother watched as the drug was slowly infused into her daughter’s tiny body as cartoons danced on a TV screen in her hospital room.

Mother whispered comforting words to daughter.

This was more than a drug. It was their hope for a normal life.

“Karen Wright worked her way through the 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.”

What was the most impactful part of your award-winning entry?
The scene at the hospital room where the baby’s mother stood by as this life-saving drug was infused into her child is most impactful. The scene had great emotional power that carried the reader into the story.

Chris Bilowich, Selena Carter, Doug Bennett, Greg Hamilton, Jesse Jones, Bill Levesque, Kyle Walker, and Rossana Passaniti.
Chris Bilowich, Selena Carter, Doug Bennett, Greg Hamilton, Jesse Jones, Bill Levesque, Kyle Walker, and Rossana Passaniti.

What is one thing you learned from this experience?
We learned how to carefully overcome the difficulty of gathering good detail and comment from a scene packed with medical professionals doing life-and-death work while not getting in anyone’s way.

What challenge did you overcome?
The biggest challenge of the story was keeping pace with a fast-moving situation as we coordinated with Londyn’s care team to ensure we were ready to document the drug infusion on a moment’s notice.

What was the biggest challenge in writing about this topic?
This was essentially a breaking news story. In words, photos and with video, the UF Health news team worked quickly as a team to document the emotions and details of an extraordinary day that delivered hope to a little girl.

Contact

Gregory Hamilton, gregoryhamilton@ufl.edu

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