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Somatic outcomes in a phase 1/2 study of weekly intraveno... | ResearchHub
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Somatic outcomes in a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in mucopolysaccharidosis type II
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Authors
Barbara Burton
•
Joseph Muenzer
20 more
•
Anna Bakardjiev
Published
February 1, 2024
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Journal
Molecular Genetics and Metabolism
Topics
Internal Medicine
Pediatrics
Biology
Internal Medicine
Medicine
Pediatrics
Genetics
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DOI
10.1016/j.ymgme.2023.107782
