title: Expanding rare disease drug trials based on shared molecular etiology.
authors.affiliation: Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.
authors.forename: Philip J
authors.initials: PJ
authors.lastname: Brooks
authors.affiliation: Office of Special Initiatives, Office of the Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.
authors.forename: Danilo A
authors.initials: DA
authors.lastname: Tagle
authors.affiliation: Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, Maryland, USA.
authors.forename: Steve
authors.initials: S
authors.lastname: Groft
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D015195:Drug Design; D006801:Humans; D058990:Molecular Targeted Therapy / Q000379:methods*; D035583:Rare Diseases / Q000145:classification / Q000188:drug therapy* / Q000378:metabolism*