Leveraging clinical metadata in Sanfilippo syndrome: A scoping review of study characteristics, data elements, outcome measures and methodologies

Molecular Genetics and Metabolism 2026

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Authors: Cara O’Neill, Jessica Haywood, Jonathan Acevedo, Rafael A. Badell‐Grau, Daniela Castillo Garcia, Patricia I. Dickson, Julie B. Eisengart, Simon Allan Jones, Inez Y. Oh, Erin Stoop, Tyler Stoop, David A. H. Whiteman
DOI: 10.1016/j.ymgme.2025.109570
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109570–109570
Publication date
1 February 2026
Language
EN