Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses

Publication date: Available online 11 June 2020Source: Molecular Genetics and MetabolismAuthor(s): Simon A. Jones, Deborah Marsden, Tony Koutsoukos, Jennifer Sniadecki, Karen Tylee, Sarah Phillippo, Emil Kakkis
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research