REGENXBIO was granted Orphan Drug Designation by the FDA for its investigational gene therapy RGX-111 for the treatment of mucopolysaccharidosis Type I (MPS I), a rare neurodegenerative disease caused by deficiency of the a-l-iduronidase
Cord blood has been proposed as an alternative stem cell source for children with HS since it has been suggested that cord blood may increase their levels of lysosomal a-L-iduronidase, which consequently may allow them to live longer with fewer complications," said lead study author jaap Jan Boelens, M.
Ninety-eight percent of the cord blood recipients whose transplanted cells were successfully engrafted had normal enzyme levels, supporting a link between high levels of lysosomal a-L-iduronidase in cord blood and suggested improvements in long-term outcomes among HS patients who receive UCB transplants.
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.
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.
Characterized by a halt in a patient's physical and mental development, MPS I 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.
Neufeld and Kakkis developed a method for producing the recombinant form of a-L-iduronidase.
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.