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Perceptions of parents of children with Sanfilippo Syndrome
Meaningfulness of clinicial and parent-reported outcome measures for use in clinical trials
Peay, H., Porter, K., Drake, E., & O'Neill, C. (2020). Perceptions of parents of children with Sanfilippo Syndrome: Meaningfulness of clinicial and parent-reported outcome measures for use in clinical trials. Value in Health, 23, S344. https://doi.org/10.1016/j.jval.2020.04.1308
PRO87 Sanfilippo Syndrome is a rare, pediatric-onset, multi-symptom disorder with no approved treatments. We report the second phase of a study to assess parent priorities regarding treatment targets and associated clinical outcome measures.
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