title: Accessing the accelerated approval pathway for rare disease therapeutics.
authors.affiliation: EveryLife Foundation for Rare Diseases, Novato, California, USA.
authors.forename: Emil D
authors.initials: ED
authors.lastname: Kakkis
authors.affiliation: EveryLife Foundation for Rare Diseases, Novato, California, USA.
authors.forename: Sara
authors.initials: S
authors.lastname: Kowalcyk
authors.affiliation: EveryLife Foundation for Rare Diseases, Novato, California, USA.
authors.forename: Max G
authors.initials: MG
authors.lastname: Bronstein
mesh_terms: D015415:Biomarkers / Q000032:analysis; D017277:Drug Approval / Q000331:legislation & jurisprudence* / Q000706:statistics & numerical data*; D006801:Humans; D035583:Rare Diseases / Q000175:diagnosis* / Q000188:drug therapy*; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015415:Biomarkers