Tenders are invited for Supply of Pharmacological agent to treat Gaucher disease caused by beta glucosidase enzyme deficiency in children younger than 18 years ,Pharmacological agent to treat Mucopolysaccharoidosis type 1 disease caused by alfa 1 Iduronidase
enzyme deficiency in children younger than 18 years
MPS I is a rare, hereditary, lysosomal storage disease that arises from a deficiency or absence of the enzyme iduronidase, which is needed to break down complex sugars produced by the body.
AGT(181, a novel, investigational enzyme replacement therapy for the treatment of both somatic and cognitive symptoms in patients with MPS I, was developed by re-engineering the enzyme iduronidase as fusion protein with an immunoglobulin G antibody targeting the insulin receptor.
MPS I is a rare, hereditary, lysosomal storage disease that arises from a deficiency or absence of the enzyme iduronidase (IDUA), which is needed to break down complex sugars produced by the body.
AGT-181 was developed by re-engineering the enzyme iduronidase (IDUA) as fusion protein with an immunoglobulin G (IgG) antibody targeting the insulin receptor.
The expanded screening for LSDs included the analysis of acid [beta]-glucocerebrosidase (ABG), [alpha]-galactosidase (GLA), [alpha]-glucosidase (GAA), acid sphingomyelinase (ASM), galactocerebrosidase (GALC), and iduronidase (IDUA) enzyme activities in the anonymized DBS samples.
7] Nonstandard abbreviations: MS/MS, tandem mass spectrometry; LSD, lysosomal storage disorder; MPS, mucopolysaccharidosis; LLE, liquid-liquid extraction; SPE, solid-phase extraction; Turboflow, turbulent flow chromatography; UHPLC, ultra-HPLC; DBS, dried blood spots; NBS, newborn screening; ABG, acid /3glucocerebrosidase; GLA, [alpha]-galactosidase; GAA, a-glucosidase; ASM, acid sphingomyelinase; GALC, galactocerebrosidase; DUA, iduronidase.
The most widely used specimens for the diagnosis of iduronidase deficiency have been homogenates of cultured fibroblasts or leukocytes.
Several microtests for the assay of iduronidase in plasma and/or leukocytes have been published (6, 7), but these methods have not been applied to DBFP.
The effect of variable incubation times on the activities of iduronidase is shown in Fig.
When samples from a control, an obligate carrier, and a Hurler patient were stored at room temperature (26[degrees]C) for the same period, the iduronidase activity decreased from of 128, 41, and 2.
The iduronidase activities of healthy individuals, newborn controls, obligate carriers, and MPS I patients are shown in Table 1.