title: A new toolkit for conducting clinical trials in rare disorders: will this dog hunt?
authors.forename: Robert G
authors.initials: RG
authors.lastname: Peterson
authors.forename: Stuart M
authors.initials: SM
authors.lastname: Macleod
authors.forename: Agnes V
authors.initials: AV
authors.lastname: Klein
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D009965:Orphan Drug Production / Q000379:methods*; D035583:Rare Diseases / Q000188:drug therapy*