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DNL310 Normalizes Primary Storage Substrates, Corrects Biomarkers of Lysosomal Dysfunction and Reduces Biomarkers of Neuronal Injury (neurofilament Light Chain) in MPS II: 2-Year Interim Analysis of a Phase 1/2 Study

Akhil Bhalla, Khishig Tsogtbaatar, Chau Tran,Ritesh Ravi,Meng Fang,Imanol Zubizarreta,Rupa Caprihan,Ajith Karunakara,Yuda Zhu, Elizabeth Sinclair, Dominique Jacquemet-Engelhart, Johannes Kast,Tony Hung,Kimberly Scearce-Levie, Matt Troyer,Peter Chin, Katia Meirelles,Anna Bakardjiev

Molecular Genetics and Metabolism(2024)

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