Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase [0.03%]
酶替代疗法治疗黏多糖贮积症VI：重组人生糖胺硫酸酯酶对患者生长和青春期发育的影响
Celeste Decker,Zi-Fan Yu,Roberto Giugliani et al.
Celeste Decker et al.
BACKGROUND AND METHODS: Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of w...
Chondroitin sulfate and growth factor signaling in the skeleton: Possible links to MPS VI [0.03%]
软骨素硫酸盐与骨骼中的生长因子信号转导：与黏多糖贮积症VI的可能联系
Tamara Alliston
Tamara Alliston
Mucopolysaccharidosis type VI (MPS VI), also called Maroteaux-Lamy syndrome, is an autosomal recessive lysosomal storage disorder caused by deficiency of a specific enzyme required for glycosaminoglycan catabolism. Deficiency in the N-acety...
Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI [0.03%]
关于黏多糖贮积症患者的骨密度评估初步报告（以MPS II和VI患者为例）
Ellen B Fung,Jo Ann Johnson,Jacqueline Madden et al.
Ellen B Fung et al.
Enzyme replacement therapy has been successful in alleviating morbidity and improving endurance in Mucopolysaccharidosis (MPS) type I, II, and VI, however little attention has been paid to the effects on bone mineralization. Brief case repo...
Growth patterns and the use of growth hormone in the mucopolysaccharidoses [0.03%]
黏多糖贮积症的生长模式及生长激素的应用
L E Polgreen,B S Miller
L E Polgreen
Short stature is characteristic of patients with mucopolysaccharidosis (MPS) diseases. For children with skeletal dysplasias, such as MPS, it is important to know the natural history of growth. An understanding of the natural growth pattern...