Divergent neurocognitive outcomes in mucopolysaccharidosis (MPS) type IIIB siblings: Tralesinidase alfa versus supportive management

Molecular Genetics and Metabolism 2026

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Authors: Irene J. Chang, Jacqueline Madden, Erin Jozwiak, Saba Sile, Ting Chang, Javier Szwarcberg, Paul Harmatz

DOI: 10.1016/j.ymgme.2025.109362

Cited by: 0

Fields of Study & Subjects

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