Ontology highlight
ABSTRACT:
SUBMITTER: Stacpoole PW
PROVIDER: S-EPMC6587967 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Stacpoole Peter W PW Shuster Jonathan J Thompson John L P Seamus JLPS Prather Robert A RA Lawson Lee Ann LA Zou Baiming B Buchsbaum Richard R Nixon Sara Jo SJ
Mitochondrion 20171110
We developed an Observer-Reported Outcome (ObsRO) survey instrument to be applied in a multicenter, placebo-controlled, crossover randomized controlled trial of dichloroacetate in children with pyruvate dehydrogenase complex deficiency. The instrument quantifies a subject's at-home level of functionality, as reported by a parent/caregiver, who were instrumental in providing the clinical descriptors and domains that formed the instrument's content. Feasibility testing of the ObsRO tool showed it ...[more]