Note that IDS-P and the substrate for a-L-iduronidase (for assay of MPS-I) (4) are structurally similar, but because they differ in the number of methylene groups in the hydrophobic linker, it should be straightforward to analyze for MPS-I and -II in the same infusion into the mass spectrometer.
a-L-Iduronidase, R-D-glucuronidase, and 2-sulfo-L-iduronate 2-sulfatase: preparation and characterization of radioactive substrates from heparin.
Recombinant human a-L-iduronidase
has been studied as an enzyme replacement therapy with follow-up data available for the first 2 years.
Food and Drug Administration (FDA) for a-L-iduronidase in mid-1999.
Results of BioMarin's pivotal trial showed that a-L-iduronidase was safe and well-tolerated and produced significant improvement in MPS-I patients.
Quantitative MRI studies demonstrated that treatment with a-L-iduronidase
was associated with a rapid 20 percent or more reduction in liver size after 6-12 weekly doses in nearly all patients, and that liver size returned to normal in the majority of patients by 26 weeks.
stated, "Definitive clinical trials are underway for BioMarin's lead enzyme replacement product, a-L-iduronidase, to treat MPS I patients.
More recently, the FDA designated BioMarin's a-L-iduronidase as a fast-track product.
and Genzyme General (NASDAQ:GENZ) intend to form a joint venture to develop and commercialize BioMarin's lead product, a-L-iduronidase, a recombinant enzyme designed to treat the genetic disorder known as mucopolysaccharidosis I (MPS I).
BioMarin initiated a pivotal clinical trial of a-L-iduronidase in January, following the collection of positive pre-clinical data.
Our data suggest that many of the clinical problems associated with MPS I patients -- such as enlarged liver and spleen, airway obstruction and joint stiffness -- may be effectively addressed with enzyme replacement therapy using recombinant a-L-iduronidase.
Characterized by a halt in the patient's physical and mental development, the disease is caused by lack of an active enzyme, a-L-iduronidase, which results in a build-up of certain carbohydrate materials in all parts of the body.