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 alpha-L-iduronidase
, which consequently may allow them to live longer with fewer complications," said lead study author Jaap Jan Boelens, MD, PhD, of the University Medical Center Utrecht, in Utrecht, Netherlands.
The result is a lack of the enzyme alpha-L-iduronidase
Cambridge, MA) announced the issuance of a third United States patent involving the lysosomal enzyme alpha-L-iduronidase
(IDUA), which is deficient in persons suffering from mucopolysaccharidosis type I (MPS I), also referred to as Hurler syndrome, Hurler-Scheie syndrome, or Scheie syndrome.
MPS I (also known as Hurler, Hurler-Scheie, and Scheie syndromes) is a life-threatening genetic disease caused by a deficiency of the enzyme alpha-L-iduronidase
Aldurazyme (laronidase) is an enzyme replacement therapy for the treatment of mucopolysaccharidosis I (MPS I), a rare, progressive and debilitating genetic disorder caused by a deficiency of the enzyme alpha-L-iduronidase
The data published in PNAS demonstrate a substantial reduction in antibody response to recombinant human alpha-L-iduronidase
(rhIDU), an enzyme that normally elicits a strong antibody response in the animal model used for these studies.
Aldurazyme is a specific form of purified recombinant human alpha-L-iduronidase
that is being investigated as an enzyme replacement therapy for MPS I, a life-threatening genetic disease for which no specific drug treatments currently exist.
announced results from studies indicating that intrathecal injection of recombinant human alpha-L-iduronidase
(rhIDU) can reduce carbohydrate storage in brain tissue in the canine model of MPS I (mucopolysaccharidosis I).
Cambridge, MA; 617-349-0271) announced that the United States Patent and Trademark Office has issued United States Patent 6,149,909 entitled, "Synthetic Alpha-L-Iduronidase
and Genetic Sequences Encoding Same.
MPS I is a rare inherited genetic disorder caused by deficient activity of the protein alpha-L-iduronidase
MPS-I is a life threatening genetic disease caused by a deficiency of the enzyme alpha-L-iduronidase
The joint venture was formed for the development and commercialization of the enzyme alpha-L-iduronidase
for the treatment of MPS-1, a chronic, debilitating genetic disease that afflicts children and leads to death before adulthood in a majority of patients.