Open AccessPediatric Orphan Drug Indications: 2010–2018
Author(s) -
Lauren Kimmel,
Rena M. Conti,
Anna Volerman,
KaoPing Chua
Publication year - 2020
Publication title -
pediatrics
Language(s) - Uncategorized
Resource type - Journals
SCImago Journal Rank - 2.611
H-Index - 345
eISSN - 1098-4275
pISSN - 0031-4005
DOI - 10.1542/peds.2019-3128
Subject(s) - orphan drug , medicine , rare disease , off label use , food and drug administration , disease , intensive care medicine , drug , approved drug , pediatrics , pharmacology , bioinformatics , biology
Orphan drug development is crucial for children, who are disproportionately affected by rare diseases. Data are lacking on the number, nature, and benefit of recently approved pediatric orphan indications.
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