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

• Príomhchruthaitheoirí: Katherine Ackerman Porter (Údar), Cara O'Neill (Údar), Elise Drake (Údar), Sara M. Andrews (Údar), Kathleen Delaney (Údar), Samantha Parker (Údar), Maria L. Escolar (Údar), Stacey Montgomery (Údar), William Moon (Údar), Carolyn Worrall (Údar), Holly L. Peay (Údar)
• Formáid: LEABHAR
• Foilsithe / Cruthaithe: SpringerOpen, 2022-04-01T00:00:00Z.
• Ábhair: article
• Rochtain ar líne: Connect to this object online.

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...