FDA’s Promising New Framework for Rare Genetic Diseases

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.

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Publication Details

Journal
JAMA Health Forum
Published
2026-03-26
DOI
https://doi.org/10.1001/jamahealthforum.2026.1227
Primary Topic
Genomics and Rare Diseases
Type
article
Field-Weighted Citation Impact
0.00
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article

FDA’s Promising New Framework for Rare Genetic Diseases

Scott Gottlieb, Maarika Kimbrell
JAMA Health Forum
Genomics and Rare Diseases
article

FDA’s Promising New Framework for Rare Genetic Diseases

Scott Gottlieb, Maarika Kimbrell
article en

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.

JAMA Health ForumVol. 7(3)
American Enterprise Institute (US), Morgan, Lewis & Bockius (United States) (US)
Zero hunger
Openalex Percentile: Top 6%
Genomics and Rare Diseases
DOIOpen Access PDFOpenAlex
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FDA’s Promising New Framework for Rare Genetic Diseases — Scott Gottlieb, Maarika Kimbrell · JAMA Health Forum (2026) | TGRS Research Map | TGRS