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Read the full May 1, 2025, GSL newsletter.
A new gene therapy to treat sickle cell disease has the potential to cure patients, and St. Louis Children’s Hospital is the first in the region—and one of the first in the nation—to offer it. The monthslong therapy program offers an alternative to other treatments, which only ease symptoms. This new therapy, approved by the FDA in 2023, is hoped to help patients not just survive, but thrive.
The Bottom Line: St. Louis is at the forefront of this lifechanging medical therapy, and will help set a global standard in treatment for sickle cell disease, which affects around 100,000 people in the U.S. alone.