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Interim Analysis of Key Clinical Outcomes from a Phase 1/2 Study of Weekly Intravenous DNL310 (Brain-Penetrant Enzyme Replacement Therapy) in MPS II

Joseph Muenzer,Barbara K. Burton,Paul Harmatz,Deepa Rajan,Simon A. Jones, Hannerieke . J. P. van den Hout,John Mitchell, Katia Meirelles,Natalie Engmann,Adam Scheller,Rupa Caprihan,Akhil Bhalla,Tony Hung, Jason Nachtigall,Imanol Zubizarreta, Sam Lu,Yuda Zhu,Peter Chin,Matt D. Troyer,Carole Ho,Wei Dong,Fabian Model

Molecular Genetics and Metabolism（2024）

Cited 0|Views24
Key words
Mucopolysaccharidoses,Enzyme Replacement Therapy
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