Enzyme replacement therapy for CLN1 batten disease that crosses the blood-brain-barrier

Molecular Genetics and Metabolism 2026 Open access

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Authors: Renuka Raman, Ben Horst, Zahra Shahrokh, Nader Hatambeygi, Maryam Zare, Magdalena Leszczyniecka, Joshua S. Harris, William A. Banks, Kim M. Hansen, Michelle A. Erickson, Sean Ekins
DOI: 10.1016/j.ymgme.2026.109733
Cited by: 2
References: 53

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Trypanosoma species research and implications

Publication details

Journal / source
Molecular Genetics and Metabolism
Publisher
Elsevier BV
Volume
147
Issue
3
Pages
109733–109733
Publication date
16 January 2026
Language
EN