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)
Abstract
Authors
I. Wiklund M. Raluy Y. Jangelind D.A.H. Whiteman A.M. Conway F. Pang D.E. Stull W.H. Chen