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Therapeutics

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How Individualized Gene and RNA Therapies Could Transform Treatment for Ultrarare Diseases

3 August 2026 12:00 AM

Personalized genetic medicines may address unique disease variants, but scientific, regulatory, ethical, and economic barriers remain substantial.

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How Individualized Gene and RNA Therapies Could Change Treatment for Ultra-Rare Diseases

26 July 2026 12:00 AM

Individualized genetic medicines may address exceptionally rare disorders, but evidence, manufacturing, safety, access, and regulation remain difficult.