Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference16 articles.
1. The mucopolysaccharidoses;Neufeld,2001
2. Management guidelines for mucopolysaccharidosis VI;Giugliani;Pediatrics,2007
3. Mucopolysaccharidosis VI;Valayannopoulos;Orphanet J. Rare Dis.,2010
4. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux–Lamy syndrome);Harmatz;J. Pediatr.,2004
5. Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement Therapy of Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): Results After 48 Weeks in a Phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase
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1. Metabolic Cardiomyopathies and Cardiac Defects in Inherited Disorders of Carbohydrate Metabolism: A Systematic Review;International Journal of Molecular Sciences;2023-05-11
2. Early enzyme replacement therapy prevents dental and craniofacial abnormalities in a mouse model of mucopolysaccharidosis type VI;Frontiers in Physiology;2022-09-21
3. Generation of a novel disease model mouse for mucopolysaccharidosis type VI via c. 252T>C human ARSB mutation knock-in;Biochemistry and Biophysics Reports;2022-09
4. Fifteen years of enzyme replacement therapy for mucopolysaccharidosis type VI (Maroteaux–Lamy syndrome): a case report;Journal of Medical Case Reports;2022-01-25
5. Glycosaminoglycans and Oligosaccharides Disorders: Glycosaminoglycans Synthesis Defects, Mucopolysaccharidoses, Oligosaccharidoses and Sialic Acid Disorders;Inborn Metabolic Diseases;2022
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