Disease characteristics, early effectiveness, and safety of vestronidase alfa for treatment of mucopolysaccharidosis VII assessed in novel disease monitoring program
Molecular Genetics and Metabolism2021Vol. 132, pp. S77–S77
Heather Lau, Antonio González-Meneses López, Maurizio Scarpa, Robert Hostutler, Lin Zhang, Betsy Malkus, Antonio Nino Ramirez, Deborah Marsden, Roberto Giugliani
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