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The FDA and Gene Therapy for Duchenne Muscular Dystrophy.

Authors :
Rind, David M.
Source :
JAMA: Journal of the American Medical Association. 5/28/2024, Vol. 331 Issue 20, p1705-1706. 2p.
Publication Year :
2024

Abstract

This Viewpoint examines the appropriateness of FDA accelerated approval of novel gene therapies to treat boys with Duchenne muscular dystrophy following clinical trials with surrogate outcomes that did not demonstrate net benefits. [ABSTRACT FROM AUTHOR]

Subjects

Subjects :
*DUCHENNE muscular dystrophy
*GENE therapy
*CLINICAL trials

Details

Language :
English
ISSN :
00987484
Volume :
331
Issue :
20
Database :
Academic Search Index
Journal :
JAMA: Journal of the American Medical Association
Publication Type :
Academic Journal
Accession number :
177610186
Full Text :
https://doi.org/10.1001/jama.2024.5613
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