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Optimizing regulatory frameworks for gene therapies in rare diseases: Challenges and solutions

1 Dec 2024

Abstract excerpt

The advent of genetic medicines and advanced diagnostics has revolutionized the treatment landscape for rare diseases and, with over 10,000 identified conditions affecting millions globally, has the potential to improve many lives. Despite this progress, only 5% of rare diseases have FDA-approved therapies, highlighting a significant unmet need. This article examines the critical need for optimizing the...

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PMID: 39720694
DOI: 10.1016/j.omtm.2024.101386

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