Molecular Genetics and Metabolism
 2022
DOI: 10.1016/j.ymgme.2021.11.133
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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 A Haroldson1,
Cj Witalisz2,
Cara Mayfield3
et al.
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