PIH40 Designing a Disease-Specific Patient Reported Outcome (PRO) for a Rare Disease: Assessing the Reliability, Validity, and Responsiveness of the Hunter Syndrome - Functional Outcomes for Clinical Understanding Scale (HS-Focus)
Ingela Wiklund(St George's, University of London), W.H. Chen, Yvonne Jangelind, Mireia Raluy(United BioSource Corporation (United Kingdom)), David Whiteman(Takeda (United States)), Francis Pang, Donald E. Stull(IQVIA (United States)), Anne Conway(University of Tennessee Health Science Center)
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