Meaningful treatment outcomes for Sanfilippo syndrome: A study of caregiver preferences and prioritization
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DOI: 10.1016/j.ymgme.2018.12.285
Abstract: Sanfilippo syndrome (MPS III) is a rare, degenerative disorder with no approved therapies. This study explored caregiver perceptions of the burden of Sanfilippo syndrome on the child and family, and meaningful benefits that are desired… read more here.
Keywords: score; progression; treatment; communication ... See more keywords