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Researchers to test gene therapy for rare diseases with a new FDA trial protocol

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Schematic of “umbrella of umbrellas” master protocol clinical trial. Credit: The American Journal of Human Genetics (2025). DOI: 10.1016/j.ajhg.2025.10.006

Earlier this year, researchers at Children’s Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to…

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