EC Number   |
Application |
Reference  |
|---|
   3.2.1.76 | pharmacology |
the enzyme is used in enzyme replacement therapy of mucopolysaccharidosis type I, MPSI, changes in hair morphology of MPSI patients treated with recombinant human enzyme, overview |
663579 |
| 3.2.1.76 | medicine |
the enzyme is a target for enzyme replacement therapy of mucopolysaccharidosis type I patients |
665139 |
| 3.2.1.76 | medicine |
the enzyme is a target for enzyme replacement therapy of lysosomal storage disorders of mucopolysaccharidosis type I patients, the therapy depends on efficient uptake of recombinant enzyme into tissues of patients |
665552 |
| 3.2.1.76 | pharmacology |
exploitation of alternative receptor systems that are independent of glycosylation but allow for efficient delivery to the lysosome |
665552 |
| 3.2.1.76 | medicine |
the recombinant enzyme is used in enzyme relacement therapy of the Hurler form of mucopolysaccharidosis type I, MPSI, the enzyme is administered intraventricularly in to 31 Sprague-Dawley rat brains showing penetration of the brain and uptak into neurons and glial cells, rat tissue distribution of human recombinant enzyme activity, overview |
666375 |
| 3.2.1.76 | medicine |
administration of a high dose of the enzyme or development of a recombinant alpha-L-iduronidase containing many mannose 6-phosphate residues is required for further improvement of enzyme replacement therapy for skeletal disorders caused by mucopolysaccharidosis I |
696554 |
| 3.2.1.76 | medicine |
commercially available recombinant human laronidase (Aldurazyme) infusion is safe and effective in stabilizing or improving pulmonary function and physical endurance. Preclinical trials of the enzyme in the canine, dog and feline model and clinicial trials with affected patients with mucopolysaccharidosis type I |
697827 |
| 3.2.1.76 | medicine |
as there is a difference in IDUA structural change between the severe mucopolysaccharidosis type I group and the attenuated one, except for a couple of mutations, structural analysis can help predict the clinical outcome of the disease |
699314 |
| 3.2.1.76 | medicine |
the benefit of enzyme replacement therapy with recombinant laronidase before hematopoietic stem cell transplantation for mucopolysaccharidosis I is linked to improvement in patient's pretransplantation condition and thus tolerance of such intensive therapy. Short-term use of laronidase is not associated with increased risk of either graft-versus-host-disease or graft failure |
699732 |
| 3.2.1.76 | medicine |
currently approved laronidase dose regimen, to treat the lysosomal storage disorder mucopolysaccharidosis type I, has similar efficacy and potentially improved safety compared to regimens using higher doses, regardless of dose frequency. The approved 0.58 mg/kg/week laronidase dose regimen provides near-maximal reductions in glycosaminoglycan storage and the best benefit-to-risk ratio. The 1.2 mg/kg every 2 weeks regimen may be an acceptable alternative for patients with difficulty receiving weekly infusions, but the long-term effects of this regimen are unknown. In general, laronidase therapy is safe and well tolerated in all treatment groups |
700229 |
