FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases

HHS.gov·Feb 23

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The FDA has launched a framework aimed at accelerating the development of individualized therapies for ultra-rare diseases. The framework may involve a new approval pathway, potentially allowing for the approval of therapies without large clinical trials. Details of the framework are still emerging, with some reports suggesting it could facilitate the development of personalized therapies, including gene editing treatments.

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View original report at HHS.gov ↗

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