Long-term administration of tralesinidase alfa enzyme replacement therapy (TA-ERT) results in profound and durable reduction of heparan sulfate (HS) and stabilization of cognitive function and cortical gray matter volume (CGMV) in patients with Sanfilippo syndrome type B (MPS IIIB)

Nicole Muschol; Mona Lindschau; İlyas Okur; Fatih Ezgu; Maria De Castro Lopez; Spyros P. Batzios; Igor Nestrašil; Saba Sile; Ting Chang; Jeffrey Zhang; Javier Szwarcberg; Paul Harmatz

doi:10.1016/j.ymgme.2025.109557

Long-term administration of tralesinidase alfa enzyme replacement therapy (TA-ERT) results in profound and durable reduction of heparan sulfate (HS) and stabilization of cognitive function and cortical gray matter volume (CGMV) in patients with Sanfilippo syndrome type B (MPS IIIB)

Molecular Genetics and Metabolism 2026

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Authors: Nicole Muschol, Mona Lindschau, İlyas Okur, Fatih Ezgu, Maria De Castro Lopez, Spyros P. Batzios, Igor Nestrašil, Saba Sile, Ting Chang, Jeffrey Zhang, Javier Szwarcberg, Paul Harmatz

DOI: 10.1016/j.ymgme.2025.109557

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: 109557–109557
Publication date: 1 February 2026
Language: EN