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Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome

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 limits u...

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Detalles Bibliográficos
Autores principales:	Porter, Katherine Ackerman, O’Neill, Cara, Drake, Elise, Andrews, Sara M., Delaney, Kathleen, Parker, Samantha, Escolar, Maria L., Montgomery, Stacey, Moon, William, Worrall, Carolyn, Peay, Holly L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer International Publishing 2022
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9038975/ https://www.ncbi.nlm.nih.gov/pubmed/35467223 http://dx.doi.org/10.1186/s41687-022-00447-w

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