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

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 save an infant named Baby KJ from dying of a ...

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Phase 3 of Singapore’s mega gene-mapping effort to enrol at least 400,000 patients

The research will support the implementation of precision medicine efforts for the population. Read more at straitstimes.com. Read more at straitstimes.com.

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Penn and Children's Hospital of Philadelphia to test gene therapy for rare diseases with new FDA protocol

New approach creates a pathway toward regulatory approval — and insurance coverage — for rare-disease treatments.