Caregivers' perspectives of the MPS IIIA (Sanfilippo sydrome type A) diagnostic journey and ways to improve for future families

Molecular Genetics and Metabolism 2026

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Authors: Kristin N. Voorhees, Joshua R. Call, Noemi Corona Cardona, Susan Holder, Luis Dante Martinez, Amber Negele, Vanessa Rangel Miller

DOI: 10.1016/j.ymgme.2025.109656

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Metabolism and Genetic Disorders Glycogen Storage Diseases and Myoclonus