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Active ingredient: Laronidase - Brands, Medical Use, Clinical Data

Brands, Medical Use, Clinical Data

Drug Category

  • Enzyme replacement

Dosage Forms

  • Solution (IV infusion)

Brands / Synonyms

Aldurazyme; Aldurazyme (Biomarin Pharmaceuticals) ; Alpha-L-iduronidase precursor

Description

Human recombinant alpha-L-iduronidase, 628 residues (mature form), produced by recombinant DNAtechnology in a Chinese hamster ovary cell line. Laronidase is a glycoprotein with a molecular weight of approximately 83 kD. The predicted amino acid sequence of the recombinant form, as well as the nucleotide sequence that encodes it, are identical to a polymorphic form of human a-L-iduronidase. It contains 6 N-linked oligosaccharide modification sites.

Indications

For the treatment of mucopolysaccharidosis

Pharmacology

Laronidase is used to treat mucopolysaccharide storage disorders (specifically mucopolysaccharidosis 1 or Hurlers syndrome) caused by deficiencies of alpha-L-iduronidase. Reduced or absent a-L-iduronidase activity results in the accumulation of the GAG substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction.

Mechanism of Action

Laronidase catalyses the hydrolysis of terminal alpha-L-iduronic acid residues of dermatan sulfate and heparin sulfate.

Absorption

Not Available

Toxicity

Not Available

Biotrnasformation / Drug Metabolism

Not Available

Contraindications

Not Available

Drug Interactions

Not Available

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