Skip to main content
Growth patterns in patients with mucopolysaccharidosis VII.
RNA analysis of the GALNS transcript reveals novel pathogenic mechanisms associated with Morquio syndrome A.
Risks of long-term port use in enzyme replacement therapy for lysosomal storage disorders.
Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients.
Pregnancy in patients with mucopolysaccharidosis: a case series.