Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials
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DOI: 10.1007/s40262-018-0721-y
Abstract: IntroductionMucopolysaccharidosis type VII (MPS VII, Sly Syndrome) is a progressive, debilitating, ultra-rare lysosomal storage disorder caused by the deficiency of β-glucuronidase (GUS), an enzyme required for breakdown of glycosaminoglycans (GAGs). Vestronidase alfa, a recombinant human… read more here.
Keywords: treatment; vii; type vii; mps vii ... See more keywords
Molecular profiling of failed endochondral ossification in mucopolysaccharidosis VII.
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DOI: 10.1016/j.bone.2019.115042
Abstract: Mucopolysaccharidosis (MPS) VII is a lysosomal storage disorder characterized by deficient activity of β-glucuronidase, leading to progressive accumulation of incompletely degraded heparan, dermatan, and chondroitin sulfate glycosaminoglycans (GAGs). Patients with MPS VII exhibit progressive skeletal… read more here.
Keywords: mucopolysaccharidosis; endochondral ossification; mps vii; ossification ... See more keywords
Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII.
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DOI: 10.1016/j.ymgme.2020.02.009
Abstract: Mucopolysaccharidosis VII (MPS VII) is a rare lysosomal storage disease characterized by a deficiency in the enzyme β-glucuronidase that has previously been successfully treated in a mouse model with enzyme replacement therapy. Here, we present… read more here.
Keywords: vestronidase; replacement therapy; vestronidase alfa; mps vii ... See more keywords
Circulatory C-type natriuretic peptide reduces mucopolysaccharidosis-associated craniofacial hypoplasia in vivo
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DOI: 10.1371/journal.pone.0277140
Abstract: Skeletal alterations in the head and neck region, such as midfacial hypoplasia, foramen magnum stenosis and spinal canal stenosis, are commonly observed in patients with mucopolysaccharidosis (MPS). However, enzyme replacement therapy (ERT), one of the… read more here.
Keywords: natriuretic peptide; hypoplasia; mps vii; type natriuretic ... See more keywords