title: Federal plan to advance targeted disease treatment unclear on benefits for children with rare diseases.
authors.forename: Deborah
authors.initials: D
authors.lastname: Levenson
mesh_terms: D002202:Capital Financing; D002648:Child; D002675:Child, Preschool; D006801:Humans; D009316:National Institutes of Health (U.S.) / Q000191:economics*; D035583:Rare Diseases / Q000191:economics / Q000235:genetics*; D014481:United States; D014486:United States Food and Drug Administration