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FDA’s Promising New Framework for Rare Genetic Diseases

Year: 2026

Type: article

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.

Source: JAMA Health Forum

DOI: 10.1001/jamahealthforum.2026.1227

Authors: Scott Gottlieb, Maarika Kimbrell

Institutions: American Enterprise Institute, Morgan, Lewis & Bockius (United States)

Language: English

Topic: Pharmaceutical Economics and Policy

Subfield: Economics and Econometrics

Field: Economics, Econometrics and Finance

Domain: Social Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $5,000

OpenAlex ID: W7140928681

FWCI:

Cites: 3

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