iduronidase การใช้

All three types result from an absence of, or insufficient levels of, the enzyme ?-L-iduronidase.
The gene is named IDUA because of its iduronidase enzyme protein product ., 52 different mutations in the IDUA gene have been shown to cause Hurler syndrome.
Glycoside hydrolase family 39 CAZY GH _ 39 comprises enzymes with several known activities; alpha-L-iduronidase ( ); beta-xylosidase ( ).
If someone is born with one normal and one defective copy of the gene ( s ) he is called a carrier and will produce less ?-L-iduronidase than an individual with two normal copies of the gene.
When Genzyme agreed to fund the development and commercialization of the drug, known as alpha-L iduronidase, BioMarin had an understanding with the FDA about the clinical testing needed to measure the drug's safety and effectiveness in MPS-1 patients.