FDA’s Promising New Framework for Rare Genetic Diseases

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Authors

Scott Gottlieb · American Enterprise Institute

Maarika Kimbrell · Morgan, Lewis & Bockius (United States)

Abstract

This JAMA Forum discusses the opportunities and challenges facing US Food and Drug Administration (FDA) new approval paths for individualized therapies to treat rare genetic diseases.

Topics & Keywords

Genomics and Rare Diseases Neurogenetic and Muscular Disorders Research Health Systems, Economic Evaluations, Quality of Life

UN Sustainable Development Goals

Zero hunger

Publication Details

Published in: JAMA Health Forum

Volume 7, Issue 3, pp. e261227-e261227

DOI: 10.1001/jamahealthforum.2026.1227

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