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Approaches to the Assessment of Clinical Benefit of Treatments for Conditions that Have Heterogeneous Symptoms and Impacts: Potential Applications in Rare Disease.
Murray LT, Howell TA, Matza LS, Eremenco S, Adams HR, Trundell D, Coons SJ; Rare Disease Subcommittee of the Patient-Reported Outcome Consortium. Murray LT, et al. Among authors: matza ls. Value Health. 2022 Nov 28:S1098-3015(22)04744-1. doi: 10.1016/j.jval.2022.11.012. Online ahead of print. Value Health. 2022. PMID: 36455827
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