Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment

por Porter, Katherine Ackerman, O’Neill, Cara, Drake, Elise, Parker, Samantha, Escolar, Maria L., Montgomery, Stacey, Moon, William, Worrall, Carolyn, Peay, Holly L.
Publicado 2020

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

por 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.
Publicado 2022