Generalized pairwise comparisons of prioritized outcomes are a powerful and patient-centric analysis of multi-domain scores.
Vaiva Deltuvaite-ThomasMickaël De BackerSamantha ParkerMarie DeneuxLynda E PolgreenCara O'NeillSamuel SalvaggioMarc BuysePublished in: Orphanet journal of rare diseases (2023)
GPC of prioritized outcomes is a statistically powerful as well as a patient-centric approach for the analysis of multi-domain scores in MPS IIIA and could be applied to other heterogeneous rare diseases.
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