Introduction：Basic information about Laronidase [usan:inn] CAS 210589-09-6, including its chemical name, molecular formula, synonyms, physicochemical properties, and safety information, etc.
Laronidase [usan:inn] Basic information
| Product Name: | Laronidase [usan:inn] |
| Synonyms: | Aldurazyme;alpha-L-Iduronidase;Ec 3.2.1.76;Human recombinant alpha-L-iduronidase;Iduronidase, alpha-L-(8-histidine) (human);Laronidase;Laronidase [usan:inn];ANTI-IDUA (CENTER) antibody produced in rabbit |
| CAS: | 210589-09-6 |
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| MW: | 0 |
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| Mol File: | Mol File |
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Laronidase [usan:inn] Chemical Properties
Safety Information
Laronidase [usan:inn] Usage And Synthesis
| Description | Mucopolysaccharidosis I (MPS I) is a rare genetic lysosomal storage disease causedby the deficiency of a-L-iduronidase, an enzyme required for the catabolism ofdermatan sulfate and heparin sulfate. The deficiency blocks the degradation of thesemucopolysaccharides, which accumulate in a variety of tissues including liver,spleen, heart and connective tissues. The clinical manifestations of MPS I caninclude progressive developmental delay, airways obstruction, hepatosplenomegaly,severe joint restriction and cardiovascular disease. There are three subtypes of MPS Idepending on its clinical severity: Hurler’s syndrome (severe), Hurler-Scheiesyndrome (moderate), and Scheie syndrome (mild). Among the existing therapies,bone marrow transplantation has been the only effective option for Hurler’ssyndrome. Laronidase was launched as an enzyme replacement therapy forthe treatment of patients with Hurler and Hurler-Scheie syndromes and patients withthe Scheie syndrome who have moderate to severe symptoms. It is a recombinantform of the human a-L-iduronidase produced by overexpression in a Chinese hamsterovary cell line. The recommended dosage regimen of laronidase is 0.58 mg/kg ofbody weight administered once weekly as an intravenous infusion. The efficacy oflaronidase was demonstrated in a 26-week, double-blind, placebo-controlled clinicaltrial by measuring improvement in pulmonary function and endurance. Thelaronidase-treated patients showed a mean increase of 4.0% in predicted forcedvital capacity (FVC) and a mean increase of 38.0 m in the distance walked in 6 minas compared with placebo-treated patients. Reductions in liver size and in urinaryglycosaminoglycan excretion were also observed. The most common adverse eventsassociated with laronidase were upper respiratory tract infection, rash and injection site reaction. |
| Originator | BioMarin (US) |
| Uses | Enzyme replacement in Mucopolysaccharidosis I (MPS I). |
| Brand name | Aldurazyme |
Laronidase [usan:inn] Preparation Products And Raw materials
