Treatment initiation with immune tolerance induction in severe mucopolysaccharidosis type II

Molecular Genetics and Metabolism 2026

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Authors: Madeline Steffensen, Caitlin Menello, Mary Casini, Can H. Ficicioglu

DOI: 10.1016/j.ymgme.2025.109627

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Virus-based gene therapy research Hemophilia Treatment and Research