HIGHLIGHTS
- who: Katherine Ackerman Porter from the Center for Genomics, Bioinformatics, and Translational Research, RTI International, Research Triangle Park, NC, USA have published the article: Caregivers` assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome, in the Journal: (JOURNAL)
- what: This study explores caregiver perspectives on the most impactful symptoms and patient-relevant clinical outcomes assessments. The aim of this study was to extend the prior research to support the selection of fit-for-purpose and patient-relevant clinical outcome assessments for prioritized symptoms. Given the concept reduction aim of the focus group, analysis . . .
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