FDA Creates Quicker Path for Gene Therapies Using Plausible Evidence for Rare Diseases

Key Entities

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  FDA Organization

  The U.S. Food and Drug Administration regulates drugs, biologics, and medical devices to ensure safety and efficacy.

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  Gene Therapies Concept

  Experimental treatments that modify genes to treat or cure diseases, particularly rare genetic disorders.

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  Rare Diseases Concept

  Conditions affecting fewer than 200,000 people in the U.S., often lacking approved treatments due to small patient numbers.

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  Plausible Evidence Concept

  Preliminary data suggesting a treatment might work, used here instead of full clinical trial results for faster approvals.