Arylsulfatase B

Arylsulfatase B 化学特性,用途語,生産方法

• 説明

  Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAGs) such as chondroitin 4-sulfate and dermatan sulfate. Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activity deficiency results in the accumulation of the GAG substrate dermatan sulfate throughout the body. This accumulation leads to widespread cellular, tissue, and organ dysfunction.In clinical studies, 1 mg/kg galsufase once-weekly for 24 weeks significantly increased baseline-adjusted mean 12 min walk distance compared to placebo, the study’s primary endpoint (+92 m; p=0.025). A positive, non-significant trend was observed in increasing performance in a 3 min stair climb test, vs. placebo (+5.7 stairs/min; p=0.053). Additionally, patients receiving galsulfase showed significant reductions in urinary GAG secretion compared to placebo, although these reductions were not sufficient to reach normal, healthy-patient levels. Adverse events associated with the use of galsulfase included abdominal pain, ear pain, chest pain, conjunctivitis, dyspnea, and pharyngitis. .

• Originator

  BioMarin (US)

• brand name

  Naglazyme

Arylsulfatase B 生産企業

Global(13)Suppliers