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Patient and Physician Perspectives Inform Clinical Trial Design for a Single Intravenous Dose of HMI-203, a Gene Therapy Candidate for Adults with Mucopolysaccharidosis Type II (MPS II, Hunter Syndrome)

Jeffrey Haroldson, Cj Witalisz, Cara Mayfield,N. Matthew Ellinwood,Leslie Urdaneta,Rick Martin, Gabriel Cohn

Molecular genetics and metabolism(2022)

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