Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome
Abstract Objectives Sanfilippo syndrome is a rare multisystem disease with no approved treatments. This study explores caregiver perspectives on the most impactful symptoms and patient-relevant clinical outcomes assessments. The pediatric onset and progressive neurodegenerative nature of Sanfilippo...
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SpringerOpen,
2022-04-01T00:00:00Z.
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