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Disease Characteristics, Effectiveness, and Safety of Vestronidase Alfa for the Treatment of Patients with Mucopolysaccharidosis VII in a Novel, Longitudinal, Multicenter Disease Monitoring Program

Roberto Giugliani,Antonio Gonzalez-Meneses,Maurizio Scarpa,Barbara Burton,Raymond Wang,Esmeralda Martins, Esmeralda Oussoren,Julia B. Hennermann,Brigitte Chabrol,Christina L. Grant,Angela Sun,Consuelo Durand,Joel Hetzer,Betsy Malkus,Deborah Marsden,J. Lawrence Merritt II

Orphanet Journal of Rare Diseases（2024）

Cited 0|Views14
Key words
Lysosomal storage disease,Non-immune hydrops fetalis,β-glucuronidase deficiency,Mucopolysaccharidosis VII,MPS VII
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