Parents of Patients Value Small Quality-of-life Benefits Not Targeted in Most Trials

Parents of Patients Value Small Quality-of-life Benefits Not Targeted in Most Trials

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Parents of Patients Value Small Quality-of-life Benefits Not Targeted in Most Trials

Parents of children with Sanfilippo syndrome place high value on therapies that provide even modest benefits to a narrower set of symptoms than most clinical trials address, according to a recent study. Understanding the impacts felt by caregivers and their treatment priorities can help determine the outcomes measured in clinical trials and the symptoms to target in future drug development. The study, “Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment,”…

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