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Naglazyme is a normal variant form of arylsulfatase B IUPAC name N-acetylgalactosamin 4-sulfatase) that is lacking in mucopolysaccharidosis VI patients.
has begun dosing patients in a Phase 2 clinical trial of Aryplase (recombinant human N-acetylgalactosamine 4-sulfatase or arylsulfatase B), an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI).
MPS VI is a life-threatening lysosomal storage disorder caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase (also known as arylsulfatase B).
(b) Arylsulfatase B: IT, 20 h; EL, 30 [micro]L of distilled water and 20 [micro]L of 15 mmol/L lead acetate in sodium acetate buffer (0.05 mol/L, pH 5.0); S, 50 [micro]L of 10 mmol/L 4MU-sulfate in sodium acetate buffer (0.05 mol/L, pH 5.0).
Activities of arylsulfatase B and [beta]-galactosidase of 18.5 and 42.1 [micro]mol/L of blood per hour (arylsulfatase B/[beta]-galactosidase ratio, 0.4), respectively, with a leukocyte count of 10 100/[mm.sup.3], changed to 9.2 and 22.2 [micro]mol/L of blood per hour (arylsulfatase B/[beta]-galactosidase ratio, 0.4), respectively, with a leukocyte count of 6000/[mm.sup.3].
(Nasdaq and SWX New Market: BMRN), Novato, Calif., has announced that the Committee for Orphan Medical Products of the European Agency for the Evaluation of Medicinal Products (COMP/EMEA) has rendered a positive opinion on the company's application for orphan drug designation in the European Community for rhASB (recombinant human arylsulfatase B), as enzyme replacement therapy for patients with Mucopolysaccharidosis (MPS) VI.