title: Pathway for approval of a gene therapy orphan product: treading new ground.
authors.forename: Barry J
authors.initials: BJ
authors.lastname: Byrne
mesh_terms: D017277:Drug Approval; D015316:Genetic Therapy; D006801:Humans; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D014481:United States; D014486:United States Food and Drug Administration
references.citation: Hum Gene Ther. 2013 Apr;24(4):355-62
references.pmid: 23517518