Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy

JAMA. 2016 Dec 13;316(22):2357-2358. doi: 10.1001/jama.2016.16437.

Authors

Aaron S Kesselheim¹, Jerry Avorn¹

Affiliation

¹ Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.

PMID: 27775756
DOI: 10.1001/jama.2016.16437

No abstract available

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Advisory Committees / statistics & numerical data
Codon, Terminator / drug effects
Codon, Terminator / genetics
Drug Approval / legislation & jurisprudence*
Dystrophin / biosynthesis*
Dystrophin / genetics
Exons / drug effects
Fees, Pharmaceutical
Humans
Morpholinos / economics
Morpholinos / therapeutic use*
Muscle, Skeletal / metabolism*
Muscular Dystrophy, Duchenne / drug therapy*
Muscular Dystrophy, Duchenne / genetics
Muscular Dystrophy, Duchenne / metabolism
Randomized Controlled Trials as Topic
Sample Size
Treatment Outcome
United States
United States Food and Drug Administration*

Substances

Codon, Terminator
Dystrophin
Morpholinos
eteplirsen