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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. 2023 Apr;26(4):547-553. doi: 10.1016/j.jval.2022.11.012. Epub 2022 Nov 28. Value Health. 2023. PMID: 36455827 Free article. Review.
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